The past decade has been one of rapid innovation in genome-editing technology. The opportunity now exists for investigators to manipulate virtually any gene in a diverse range of cell types and organisms with targeted nucleases designed with sequence-specific DNA-binding domains. The rapid development of the field has allowed for highly efficient, precise, and now cost-effective means by which to generate human and animal models of disease using these technologies. This review will outline the recent development of genome-editing technology, culminating with the use of CRISPR-Cas9 to generate novel mammalian models of disease. While the road to using this same technology for treatment of human disease is long, the pace of innovation over the past five years and early successes in model systems build anticipation for this prospect.
Rajat M. Gupta, Kiran Musunuru
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Therapeutic Targeting in Ovarian Cancer: Nano-Enhanced CRISPR/Cas9 Gene Editing and Drug Combination Therapy
Kim HK, Cheong H, Kim MY, Jin HE |
International Journal of Nanomedicine | 2025 |
Lymphoblastoid and Jurkat cell lines are useful surrogate in developing a CRISPR-Cas9 method to correct leukocyte adhesion deficiency genomic defect
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Frontiers in Bioengineering and Biotechnology | 2025 |
Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies
Haque US, Yokota T |
Degenerative Neurological and Neuromuscular Disease | 2025 |
Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects
Cetin B, Erendor F, Eksi YE, Sanlioglu AD, Sanlioglu S |
Expert Reviews in Molecular Medicine | 2025 |
Powering new therapeutics with precision mitochondrial editing.
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Nature biotechnology | 2025 |
Protein Tyrosine Phosphatase Studies in Zebrafish.
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Methods in molecular biology (Clifton, N.J.) | 2024 |
Genetic improvement in edible fish: status, constraints, and prospects on CRISPR-based genome engineering
Puthumana J, Chandrababu A, Sarasan M, Joseph V, Singh IS |
3 Biotech | 2024 |
CRISPR/Cas9 as a Mutagenic Factor
Shumega AR, Pavlov YI, Chirinskaite AV, Rubel AA, Inge-Vechtomov SG, Stepchenkova EI |
International journal of molecular sciences | 2024 |
Lipofection-Based Delivery of CRISPR/Cas9 Ribonucleoprotein for Gene Editing in Male Germline Stem Cells.
Obermeier M, Rogiers V, Vanhaecke T, Baert Y |
Methods in molecular biology (Clifton, N.J.) | 2024 |
Precise genome-editing in human diseases: mechanisms, strategies and applications
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Heliyon | 2024 |
Recent Advances in Tomato Gene Editing.
Larriba E, Yaroshko O, Pérez-Pérez JM |
International journal of molecular sciences | 2024 |
Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.
Leal AF, Herreno-Pachón AM, Benincore-Flórez E, Karunathilaka A, Tomatsu S |
International journal of molecular sciences | 2024 |
Editorial: First Regulatory Approvals for CRISPR-Cas9 Therapeutic Gene Editing for Sickle Cell Disease and Transfusion-Dependent β-Thalassemia.
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Genetic Manipulation Approaches to Enhance the Clinical Application of NK Cell-Based Immunotherapy.
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European journal of pharmaceutics and biopharmaceutics : official journal of Arbeitsgemeinschaft fur Pharmazeutische Verfahrenstechnik e.V | 2024 |
CRISPR/Cas9 genome editing of CCR5 combined with C46 HIV-1 fusion inhibitor for cellular resistant to R5 and X4 tropic HIV-1.
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Scientific Reports | 2024 |
Multiplex CRISPR-Cas Genome Editing: Next-Generation Microbial Strain Engineering
Lim SR, Lee SJ |
Journal of Agricultural and Food Chemistry | 2024 |
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Nature reviews. Neuroscience | 2024 |
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Science and Public Policy | 2024 |
Role of CRISPR-Cas systems and anti-CRISPR proteins in bacterial antibiotic resistance.
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Heliyon | 2024 |
Novel approaches for HTLV-1 therapy: innovative applications of CRISPR-Cas9
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Revista do Instituto de Medicina Tropical de São Paulo | 2024 |
Rice Promoter Editing: An Efficient Genetic Improvement Strategy
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Rice | 2024 |
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Nature communications | 2024 |
A platform to deliver single and bi-specific Cas9/guide RNA to perturb genes in vitro and in vivo
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Current gene therapy | 2024 |
Reporter Alleles in hiPSCs: Visual Cues on Development and Disease.
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International journal of molecular sciences | 2024 |
Allogeneic chimeric antigen receptor cell therapies for cancer: progress made and remaining roadblocks.
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Nature reviews. Clinical oncology | 2024 |
Enhancing Cell Line Stability by CRISPR/Cas9-Mediated Site-Specific Integration Based on Histone Modifications.
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Methods in molecular biology (Clifton, N.J.) | 2024 |
Understanding the HPV associated cancers: A comprehensive review.
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Molecular biology reports | 2024 |
Optimizing cancer treatment: the synergistic potential of CAR-T cell therapy and CRISPR/Cas9
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Frontiers in Immunology | 2024 |
Chasing shadows: Investigating X chromosome mediation in late-onset Alzheimer’s disease
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Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine
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Frontiers in Genome Editing | 2024 |
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Exploring Advanced CRISPR Delivery Technologies for Therapeutic Genome Editing
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Small Science | 2024 |
Recent Advances in Genome-Engineering Strategies
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Genes & development | 2023 |
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Human papillomavirus in the setting of immunodeficiency: Pathogenesis and the emergence of next-generation therapies to reduce the high associated cancer risk
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Frontiers in immunology | 2023 |
Principles of Zebrafish Nephron Segment Development.
Nguyen TK, Petrikas M, Chambers BE, Wingert RA |
Journal of Developmental Biology | 2023 |
Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing.
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Drug Delivery and Translational Research | 2023 |
Unclasping potentials of genomics and gene editing in chickpea to fight climate change and global hunger threat
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Frontiers in Genetics | 2023 |
CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
Pinjala P, Tryphena KP, Prasad R, Khatri DK, Sun W, Singh SB, Gugulothu D, Srivastava S, Vora L |
Biomaterials Research | 2023 |
Site-specific gene knock-in and bacterial phytase gene expression in Chlamydomonas reinhardtii via Cas9 RNP-mediated HDR
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Frontiers in Plant Science | 2023 |
CRISPR/Cas-mediated genome editing in mice for the development of drug delivery mechanism.
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Molecular Biology Reports | 2023 |
What did CRISPR-Cas9 accomplish in its first 10 years?
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Biochemia Medica | 2023 |
Review of recent advances in post-harvest techniques for tropical cut flowers and future prospects: Heliconia as a case-study.
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Frontiers in Plant Science | 2023 |
Therapeutic Applications of CRISPR/Cas9 Gene Editing Technology for the Treatment of Ocular Diseases
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The FEBS journal | 2023 |
Progress and Perspective of CRISPR-Cas9 Technology in Translational Medicine.
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2023 | |
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2023 | |
En route towards a personalized medicine approach: Innovative therapeutic modalities for connective tissue disorders
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Matrix biology : journal of the International Society for Matrix Biology | 2023 |
Applications of Genome Editing Technologies in CAD Research and Therapy with a Focus on Atherosclerosis
Mak MC, Gurung R, Foo RS |
International journal of molecular sciences | 2023 |
Genome-wide CRISPR screens and their applications in infectious disease
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2023 | |
Advances in Recombinant Adeno-Associated Virus Vectors for Neurodegenerative Diseases
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Biomedicines | 2023 |
Illuminating the oral microbiome: cellular microbiology
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FEMS Microbiology Reviews | 2023 |
CRISPR-Cas Technology as a Revolutionary Genome Editing tool: Mechanisms and Biomedical Applications.
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Iranian Biomedical Journal | 2023 |
CRISPR-Cas Technology as a Revolutionary Genome Editing tool: Mechanisms and Biomedical Applications.
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Iranian Biomedical Journal | 2023 |
Gene Editing in Hematopoietic Stem Cells.
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Advances in experimental medicine and biology | 2023 |
Genetic Kidney Diseases (GKDs) Modeling Using Genome Editing Technologies
F Gómez-García, R Martínez-Pulleiro, N Carrera, C Allegue, M Garcia-Gonzalez |
Cells | 2022 |
CRISPR Technology in Cancer Diagnosis and Treatment: Opportunities and Challenges
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Biochemical Genetics | 2022 |
CRISPR Therapeutics for Duchenne Muscular Dystrophy
E Erkut, T Yokota |
International journal of molecular sciences | 2022 |
Challenges of CRISPR-Based Gene Editing in Primary T Cells
A Rezalotfi, L Fritz, R Förster, B Bošnjak |
International journal of molecular sciences | 2022 |
Gene Editing with CRISPR/Cas Methodology and Thyroid Cancer: Where Are We?
C Fuziwara, D de Mello, E Kimura |
Cancers | 2022 |
The Basis and Promise of Programmable RNA Editing and Modification
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Frontiers in Genetics | 2022 |
Utilization of CRISPR-Mediated Tools for Studying Functional Genomics in Hematological Malignancies: An Overview on the Current Perspectives, Challenges, and Clinical Implications
M Solayappan, A Azlan, K Khor, M Yik, M Khan, N Yusoff, E Moses |
Frontiers in Genetics | 2022 |
CRISPR based therapeutics: a new paradigm in cancer precision medicine
S Das, S Bano, P Kapse, G Kundu |
Molecular Cancer | 2022 |
CRISPR/Cas: A New Tool in the Research of Telomeres and Telomerase as Well as a Novel Form of Cancer Therapy
M Porika, R Tippani, G Saretzki |
International journal of molecular sciences | 2022 |
Modified Gene Editing Systems: Diverse Bioengineering Tools and Crop Improvement
G Zhu, H Zhu |
Frontiers in Plant Science | 2022 |
Multiple Gene Transfer and All-In-One Conditional Knockout Systems in Mouse Embryonic Stem Cells for Analysis of Gene Function
T Suzuki, S Takagi, T Hara |
Frontiers in Cell and Developmental Biology | 2022 |
Enhancing HR Frequency for Precise Genome Editing in Plants
H Chen, M Neubauer, J Wang |
Frontiers in Plant Science | 2022 |
Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment
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Biology | 2022 |
Closing the Door with CRISPR: Genome Editing of CCR5 and CXCR4 as a Potential Curative Solution for HIV
Freen-van Heeren JJ |
Bio/Technology | 2022 |
Stimuli-responsive nanoformulations for CRISPR-Cas9 genome editing
Fang T, Cao X, Ibnat M, Chen G |
Journal of nanobiotechnology | 2022 |
Plasmopara viticola the Causal Agent of Downy Mildew of Grapevine: From Its Taxonomy to Disease Management
Koledenkova K, Esmaeel Q, Jacquard C, Nowak J, Clément C, Ait Barka E |
Frontiers in microbiology | 2022 |
Genome Editing Targets for Improving Nutrient Use Efficiency and Nutrient Stress Adaptation
Sathee L, Jagadhesan B, Pandesha PH, Barman D, Adavi B S, Nagar S, Krishna GK, Tripathi S, Jha SK, Chinnusamy V |
Frontiers in Genetics | 2022 |
Lipofection of Non-integrative CRISPR/Cas9 Ribonucleoproteins in Male Germline Stem Cells: A Simple and Effective Knockout Tool for Germline Genome Engineering
Obermeier M, Vadolas J, Verhulst S, Goossens E, Baert Y |
Frontiers in Cell and Developmental Biology | 2022 |
Advancements in Genomic and Behavioral Neuroscience Analysis for the Study of Normal and Pathological Brain Function
Baratta AM, Brandner AJ, Plasil SL, Rice RC, Farris SP |
Frontiers in molecular neuroscience | 2022 |
Zebrafish Modeling of Autism Spectrum Disorders, Current Status and Future Prospective
Tayanloo-Beik A, Hamidpour SK, Abedi M, Shojaei H, Tavirani MR, Namazi N, Larijani B, Arjmand B |
Frontiers in Psychiatry | 2022 |
Strategies to Improve the Safety of iPSC-Derived β Cells for β Cell Replacement in Diabetes
Pellegrini S, Zamarian V, Sordi V |
Transplant International | 2022 |
The Bibliometric Landscape of Gene Editing Innovation and Regulation in the Worldwide
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Cells | 2022 |
Inhibition of HIV-1 replication using the CRISPR/cas9-no NLS system as a prophylactic strategy
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Heliyon | 2022 |
Genome editing in cancer: Challenges and potential opportunities
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Bioactive Materials | 2022 |
Advances in CRISPR therapeutics.
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Nature reviews. Nephrology | 2022 |
Application of advanced technology in traditional Chinese medicine for cancer therapy.
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Frontiers in pharmacology | 2022 |
Integration of CRISPR/Cas9 with artificial intelligence for improved cancer therapeutics
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Journal of Translational Medicine | 2022 |
Virus-induced gene silencing for in planta validation of gene function in cucurbits
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Plant physiology | 2022 |
From a Single Cell to a Whole Human Liver: Disease Modeling and Transplantation.
Motomura T, Faccioli LAP, Diaz-Aragon R, Kocas-Kilicarslan ZN, Haep N, Florentino RM, Amirneni S, Cetin Z, Peri BS, Morita K, Ostrowska A, Takeishi K, Soto-Gutierrez A, Tafaleng EN |
Seminars in liver disease | 2022 |
Generation of Endogenous Promoter-Driven Luciferase Reporter System Using CRISPR/Cas9 for Investigating Transcriptional Regulation of the Core Clock Gene BMAL1
Sun C, Li C, Liu W, Schiöth HB |
Biomedicines | 2022 |
Advances in CRISPR Delivery Methods: Perspectives and Challenges
Demirci S, Essawi K, Germino-Watnick P, Liu X, Hakami W, Tisdale JF |
The CRISPR journal | 2022 |
The recent advances and future perspectives of genetic compensation studies in the zebrafish model
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Genes & Diseases | 2022 |
Immunotherapy and Gene Therapy for Oncoviruses Infections: A Review
NA de Almeida, CR de Almeida Ribeiro, JV Raposo, VS de Paula |
Viruses | 2021 |
The comparison of ZFNs, TALENs, and SpCas9 by GUIDE-seq in HPV-targeted gene therapy
Z Cui, H Liu, H Zhang, Z Huang, R Tian, L Li, W Fan, Y Chen, L Chen, S Zhang, BC Das, K Severinov, II Hitzeroth, PR Debata, Z Jin, J Liu, Z Huang, W Xie, H Xie, B Lang, J Ma, H Weng, X Tian, Z Hu |
Molecular Therapy — Nucleic Acids | 2021 |
Baboon Envelope Pseudotyped “Nanoblades” Carrying Cas9/gRNA Complexes Allow Efficient Genome Editing in Human T, B, and CD34+ Cells and Knock-in of AAV6-Encoded Donor DNA in CD34+ Cells
A Gutierrez-Guerrero, MJ Recalde, PE Mangeot, C Costa, O Bernadin, S Périan, F Fusil, G Froment, A Martinez-Turtos, A Krug, F Martin, K Benabdellah, EP Ricci, S Giovannozzi, R Gijsbers, E Ayuso, FL Cosset, E Verhoeyen |
2021 | |
Genetics, pathogenesis and therapeutic developments for Usher syndrome type 2
M Stemerdink, B García-Bohórquez, R Schellens, G Garcia-Garcia, EV Wijk, JM Millan |
Human Genetics | 2021 |
Establishment of a Genome Editing Tool Using CRISPR-Cas9 in Chlorella vulgaris UTEX395
J Kim, KS Chang, S Lee, ES Jin |
International journal of molecular sciences | 2021 |
Utility of Zebrafish Models of Acquired and Inherited Long QT Syndrome
KE Simpson, R Venkateshappa, ZK Pang, S Faizi, GF Tibbits, TW Claydon |
Frontiers in physiology | 2021 |
Cardiomyocyte Death and Genome-Edited Stem Cell Therapy for Ischemic Heart Disease
HM Cho, JY Cho |
Stem Cell Reviews and Reports | 2021 |
Future Approaches for Treating Chronic Myeloid Leukemia: CRISPR Therapy
E Vuelta, I García-Tuñón, P Hernández-Carabias, L Méndez, M Sánchez-Martín |
Biology : open access journal | 2021 |
Genome-editing approaches and applications: a brief review on CRISPR technology and its role in cancer
N Siva, S Gupta, A Gupta, JN Shukla, B Malik, N Shukla |
3 Biotech | 2021 |
CRISPR: A new paradigm of theranostics
N Yadav, J Narang, AK Chhillar, JS Rana |
Nanomedicine : nanotechnology, biology, and medicine | 2021 |
CRISPR Gene-Editing Models Geared Toward Therapy for Hereditary and Developmental Neurological Disorders
PK Wong, FC Cheah, SE Syafruddin, MA Mohtar, N Azmi, PY Ng, EW Chua |
Frontiers in Pediatrics | 2021 |
New insights on CRISPR/Cas9-based therapy for breast Cancer
H Sabit, S Abdel-Ghany, H Tombuloglu, E Cevik, A Alqosaibi, F Almulhim, A Al-Muhanaa |
Genes and Environment | 2021 |
Patch selection by bumble bees navigating discontinuous landscapes
FP Fragoso, Q Jiang, MK Clayton, J Brunet |
Scientific Reports | 2021 |
Attitudes of Indonesian Medical Doctors and Medical Students Toward Genome Editing
SN Izzah, D Setyanto, N Hasanatuludhhiyah, DN Indiastuti, Z Nasution, A d'Arqom |
Journal of multidisciplinary healthcare | 2021 |
Discovering antiviral restriction factors and pathways using genetic screens
CE Jones, WS Tan, F Grey, DJ Hughes |
Journal of General Virology | 2021 |
Applications and Major Achievements of Genome Editing in Vegetable Crops: A Review
YC Kim, Y Kang, EY Yang, MC Cho, R Schafleitner, JH Lee, S Jang |
Frontiers in Plant Science | 2021 |
Germline genome modification through novel political, ethical, and social lenses
V Xafis, GO Schaefer, MK Labude, Y Zhu, S Holm, RS Foo, PS Lai, R Chadwick, HT Greely |
PLoS genetics | 2021 |
From Descriptive to Functional Genomics of Leukemias Focusing on Genome Engineering Techniques
B Balla, F Tripon, C Banescu |
International journal of molecular sciences | 2021 |
Synthetic polycistronic sequences in eukaryotes
X Wang, MA Marchisio |
Synthetic and Systems Biotechnology | 2021 |
Therapeutic Genome Editing and In Vivo Delivery
AC Ramirez-Phillips, D Liu |
The AAPS Journal | 2021 |
Will CRISPR-Cas9 Have Cards to Play Against Cancer? An Update on its Applications
PS Daisy, KS Shreyas, TS Anitha |
Molecular Biotechnology | 2021 |
Application of Genome Editing in Tomato Breeding: Mechanisms, Advances, and Prospects
H Salava, S Thula, V Mohan, R Kumar, F Maghuly |
International journal of molecular sciences | 2021 |
Application of CRISPR–Cas9 in plant–plant growth-promoting rhizobacteria interactions for next Green Revolution
S Singh, W Ramakrishna |
3 Biotech | 2021 |
CRISPR and KRAS: a match yet to be made
G Bender, RF Yamaci, B Taneri |
Journal of Biomedical Science | 2021 |
CRISPR Gene Editing in Lipid Disorders and Atherosclerosis: Mechanisms and Opportunities
HE Walker, M Rizzo, Z Fras, B Jug, M Banach, PE Penson |
Metabolites | 2021 |
CRISPER/CAS System, a Novel Tool of Targeted Therapy of Drug-Resistant Lung Cancer
Akbari Kordkheyli V, Rashidi M, Shokri Y, Fallahpour S, Variji A, Nabipour Ghara E, Hosseini SM |
Advanced pharmaceutical bulletin | 2021 |
Genome Editing in Medicine: Tools and Challenges
Petraitytė G, Preikšaitienė E, Mikštienė V |
Acta medica Lituanica | 2021 |
CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics
Bhardwaj S, Kesari KK, Rachamalla M, Mani S, Ashraf GM, Jha SK, Kumar P, Ambasta RK, Dureja H, Devkota HP, Gupta G, Chellappan DK, Singh SK, Dua K, Ruokolainen J, Kamal MA, Ojha S, Jha NK |
Journal of Advanced Research | 2021 |
Correction of Muscular Dystrophies by CRISPR Gene Editing
Francesco Chemello, Rhonda Bassel-Duby, Eric N. Olson |
Journal of Clinical Investigation | 2020 |
Sharpening the Molecular Scissors: Advances in Gene-Editing Technology
M Broeders, P Herrero-Hernandez, MP Ernst, AT van der Ploeg, WW Pijnappel |
iScience | 2020 |
Lysosomal storage diseases: current therapies and future alternatives
AF Leal, AJ Espejo-Mojica, OF Sánchez, CM Ramírez, LH Reyes, JC Cruz, CJ Alméciga-Díaz |
Journal of Molecular Medicine | 2020 |
High level of fetal-globin reactivation by designed transcriptional activator-like effector
J Zhan, MJ Irudayam, Y Nakamura, R Kurita, AW Nienhuis |
Blood Advances | 2020 |
Novel genetic therapeutic approaches for modulating the severity of β‑thalassemia (Review)
F Amjad, T Fatima, T Fayyaz, M Khan, M Qadeer |
Biomedical Reports | 2020 |
Compensation of Disabled Organogeneses in Genetically Modified Pig Fetuses by Blastocyst Complementation
H Matsunari, M Watanabe, K Hasegawa, A Uchikura, K Nakano, K Umeyama, H Masaki, S Hamanaka, T Yamaguchi, M Nagaya, R Nishinakamura, H Nakauchi, H Nagashima |
Stem Cell Reports | 2020 |
The CRISP(Y) Future of Pediatric Soft Tissue Sarcomas
S Pomella, R Rota |
Frontiers in Chemistry | 2020 |
Human T cell glycosylation and implications on immune therapy for cancer
ED Bousser, L Meuris, N Callewaert, N Festjens |
Human Vaccines & Immunotherapeutics | 2020 |
Multigene CRISPR/Cas9 genome editing of hybrid proline rich proteins (HyPRPs) for sustainable multi-stress tolerance in crops: the review of a promising approach
B Saikia, S Singh, J Debbarma, N Velmurugan, H Dekaboruah, KP Arunkumar, C Chikkaputtaiah |
Physiology and Molecular Biology of Plants | 2020 |
How CRISPR-Cas System Could Be Used to Combat Antimicrobial Resistance P Gholizadeh, Ş Köse, S Dao, K Ganbarov, A Tanomand, T Dal, M Aghazadeh, R Ghotaslou, MA Rezaee, B Yousefi, HS Kafil |
Infection and drug resistance | 2020 |
The clinical potential of gene editing as a tool to engineer cell-based therapeutics
C Ashmore-Harris, GO Fruhwirth |
Clinical and Translational Medicine | 2020 |
Progress and Challenges in the Improvement of Ornamental Plants by Genome Editing
CH Ahn, M Ramya, HR An, PM Park, YJ Kim, SY Lee, S Jang |
Plants | 2020 |
Advance genome editing technologies in the treatment of human diseases: CRISPR therapy (Review)
M Alagoz, N Kherad |
International journal of molecular medicine | 2020 |
Development of a Self-Restricting CRISPR-Cas9 System to Reduce Off-Target Effects
H Wang, H Lu, Y Lei, C Gong, Z Chen, Y Luan, Q Li, Y Jian, H Wang, F Wu, C Tao, H Shen, H Bo, H Shao, W Zhang |
Molecular Therapy — Methods & Clinical Development | 2020 |
Site-Specific Gene Knock-Out and On-Site Heterologous Gene Overexpression in Chlamydomonas reinhardtii via a CRISPR-Cas9-Mediated Knock-in Method
J Kim, S Lee, K Baek, ES Jin |
Frontiers in Plant Science | 2020 |
Nucleic Acid Therapy for β-Thalassemia A d'Arqom |
Biologics : targets & therapy | 2020 |
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock
R Martier, P Konstantinova |
Frontiers in neuroscience | 2020 |
Base editing: advances and therapeutic opportunities
EM Porto, AC Komor, IM Slaymaker, GW Yeo |
Nature Reviews Drug Discovery | 2020 |
A versatile toolkit for CRISPR-Cas13-based RNA manipulation in Drosophila
N Huynh, N Depner, R Larson, K King-Jones |
Genome biology | 2020 |
Genome Editing by CRISPR-Cas: A Game Change in the Genetic Manipulation of Chlamydomonas
M Ghribi, SB Nouemssi, F Meddeb-Mouelhi, I Desgagné-Penix |
Life Sciences | 2020 |
CRISPR FokI Dead Cas9 System: Principles and Applications in Genome Engineering
M Saifaldeen, DE Al-Ansari, D Ramotar, M Aouida |
Cells | 2020 |
Various Aspects of a Gene Editing System—CRISPR–Cas9
E Janik, M Niemcewicz, M Ceremuga, L Krzowski, J Saluk-Bijak, M Bijak |
International journal of molecular sciences | 2020 |
Novel insights into gene therapy in the cornea
RR Mohan, LM Martin, NR Sinha |
Experimental Eye Research | 2020 |
Targeting of cholera toxin A (ctxA) gene by zinc finger nuclease: pitfalls of using gene editing tools in prokaryotes
N Hosseini, H Khanahmad, BN Esfahani, M Bandehpour, L Shariati, N Zahedi, B Kazemi |
Research in Pharmaceutical Sciences | 2020 |
Gene therapy of hematological disorders: current challenges
AM Al-Saif |
Gene Therapy | 2019 |
Programmable Molecular Scissors: Applications of a New Tool for Genome Editing in Biotech
SK Saha, FK Saikot, S Rahman, MA Jamal, SM Rahman, SM Islam, KH Kim |
Molecular Therapy — Nucleic Acids | 2019 |
hiPSCs in cardio-oncology: deciphering the genomics
EA Pinheiro, KA Fetterman, PW Burridge |
Cardiovascular Research | 2019 |
Targeting Telomeres and Telomerase: Studies in Aging and Disease Utilizing CRISPR/Cas9 Technology
A Brane, T Tollefsbol |
Cells | 2019 |
Use of Genome Editing Tools in Environmental Health Research
JE Rager, C Carberry, RC Fry |
Current Opinion in Toxicology | 2019 |
An update on the tools for creating transgenic animal models of human diseases – focus on atherosclerosis
AS Volobueva, AN Orekhov, AV Deykin |
Brazilian journal of medical and biological research = Revista brasileira de pesquisas medicas e biologicas / Sociedade Brasileira de Biofisica ... [et al.] | 2019 |
New Human Chromosomal Sites with “Safe Harbor” Potential for Targeted Transgene Insertion
S Pellenz, M Phelps, W Tang, BT Hovde, RB Sinit, W Fu, H Li, E Chen, RJ Monnat |
Human Gene Therapy | 2019 |
Public attitudes towards genetically modified polled cattle
E McConnachie, MJ Hötzel, JA Robbins, A Shriver, DM Weary, MA von Keyserlingk, IA Olsson |
PloS one | 2019 |
An Overview Of The Crispr-Based Genomic- And Epigenome-Editing System: Function, Applications, And Challenges
SB Qomi, A Asghari, M Mojarrad |
Advanced Biomedical Research | 2019 |
Transgenerational Perpetuation of CHS Gene Expression and DNA Methylation Status Induced by Short Oligodeoxynucleotides in Flax (Linum usitatissimum)
M Dzialo, J Szopa, A Hnitecka, M Zuk |
International journal of molecular sciences | 2019 |
Sheep and Goat Genome Engineering: From Random Transgenesis to the CRISPR Era
P Kalds, S Zhou, B Cai, J Liu, Y Wang, B Petersen, T Sonstegard, X Wang, Y Chen |
Frontiers in Genetics | 2019 |
Era of Genomic Medicine: A Narrative Review on CRISPR Technology as a Potential Therapeutic Tool for Human Diseases
OW Kotagama, CD Jayasinghe, T Abeysinghe |
BioMed Research International | 2019 |
Gene surgery: Potential applications for human diseases
Ayman El-Kenawy, Bachir Benarba, Adriana Freitas Neves, Thaise Gonçalves de Araujo, Bee Ling Tan, Adel Gouri |
EXCLI J | 2019 |
The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements
P Patsali, C Mussolino, P Ladas, A Floga, A Kolnagou, S Christou, M Sitarou, MN Antoniou, T Cathomen, CW Lederer, M Kleanthous |
Journal of Clinical Medicine | 2019 |
The potential of genomics for restoring ecosystems and biodiversity
MF Breed, PA Harrison, C Blyth, M Byrne, V Gaget, NJ Gellie, SV Groom, R Hodgson, JG Mills, TA Prowse, DA Steane, JJ Mohr |
Nature Reviews Genetics | 2019 |
Climatic changes and potatoes: How can we cope with the abiotic stresses?
T Handayani, SA Gilani, KN Watanabe |
Breeding Science | 2019 |
Zebrafish In Vivo Models of Cancer and Metastasis
KR Astell, D Sieger |
Cold Spring Harbor Perspectives in Medicine | 2019 |
Synthetic microbial consortia for biosynthesis and biodegradation: promises and challenges
S Che, Y Men |
Journal of Industrial Microbiology & Biotechnology | 2019 |
Evaluation of the CRISPR/Cas9 directed mutant TP53 gene repairing effect in human prostate cancer cell line PC-3
MB Batır, E Şahin, FS Çam |
Molecular Biology Reports | 2019 |
Gene Editing and Gene-Based Therapeutics for Cardiomyopathies
JC Ohiri, EM McNally |
Heart Failure Clinics | 2018 |
Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency
HH Abdul-Razak, CJ Rocca, SJ Howe, ME Alonso-Ferrero, J Wang, R Gabriel, CC Bartholomae, CH Gan, MI Garín, A Roberts, MP Blundell, V Prakash, FJ Molina-Estevez, J Pantoglou, G Guenechea, MC Holmes, PD Gregory, C Kinnon, C von Kalle, M Schmidt, JA Bueren, AJ Thrasher, RJ Yáñez-Muñoz |
Scientific Reports | 2018 |
Delivering CRISPR: a review of the challenges and approaches
CA Lino, JC Harper, JP Carney, JA Timlin |
Drug Delivery | 2018 |
Knocking out Ornithine Decarboxylase Antizyme 1 (OAZ1) Improves Recombinant Protein Expression in the HEK293 Cell Line
L Abaandou, J Shiloach |
Medical Sciences | 2018 |
Zebrafish as a model for kidney function and disease
P Outtandy, C Russell, R Kleta, D Bockenhauer |
Pediatric Nephrology | 2018 |
Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber’s Hereditary Optic Neuropathy
SE Ratican, A Osborne, KR Martin |
Neural plasticity | 2018 |
CRISPR/Cas9 genome surgery for retinal diseases
CL Xu, KS Park, SH Tsang |
Drug discovery today. Technologies | 2018 |
The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Alzheimer's Disease
TT Rohn, N Kim, NF Isho, JM Mack |
Journal of Alzheimer's disease & Parkinsonism | 2018 |
Evolution of the SPX gene family in plants and its role in the response mechanism to phosphorus stress
N Liu, W Shang, C Li, L Jia, X Wang, G Xing, WM Zheng |
Open Biology | 2018 |
In vivo epigenome editing and transcriptional modulation using CRISPR technology
CH Lau, Y Suh |
Transgenic Research | 2018 |
Genome Editing: A New Horizon for Oral and Craniofacial Research
N Yu, J Yang, Y Mishina, WV Giannobile |
Journal of dental research | 2018 |
A Drosophila CRISPR/Cas9 Toolkit for Conditionally Manipulating Gene Expression in the Prothoracic Gland as a Test Case for Polytene Tissues
N Huynh, J Zeng, W Liu, K King-Jones |
G3: Genes|Genomes|Genetics | 2018 |
Current progress in CRISPR‐based diagnostic platforms
K Khambhati, G Bhattacharjee, V Singh |
Journal of Cellular Biochemistry | 2018 |
The road less traveled: strategies to enhance the frequency of homology-directed repair (HDR) for increased efficiency of CRISPR/Cas-mediated transgenesis
Devkota S |
BMB Reports | 2018 |
Atherogenesis and Metabolic Dysregulation in LDL Receptor Knockout Rats
Srinivas Dwarakanath Sithu, Marina V. Malovichko, Krista A Riggs, Nalinie S Wickramasinghe, Millicent G Winner, Abhinav Agarwal, Rihab E Hamed-Berair, Anuradha Kalani, Daniel W Riggs, Aruni Bhatnagar, Sanjay Srivastava |
JCI Insight | 2017 |
Transcriptional control of chondrocyte specification and differentiation
CF Liu, WE Samsa, G Zhou, V Lefebvre |
Seminars in Cell & Developmental Biology | 2017 |
The Impact of CRISPR/Cas9 Technology on Cardiac Research: From Disease Modelling to Therapeutic Approaches
BM Motta, PP Pramstaller, AA Hicks, A Rossini |
Stem Cells International | 2017 |
Genome Editing and Muscle Stem Cells as a Therapeutic Tool for Muscular Dystrophies
V Pini, JE Morgan, F Muntoni, HC ONeill |
Current Stem Cell Reports | 2017 |
CRISPR/Cas9: Transcending the Reality of Genome Editing
S Chira, D Gulei, A Hajitou, AA Zimta, P Cordelier, I Berindan-Neagoe |
Molecular Therapy - Nucleic Acids | 2017 |
Frameshift indels introduced by genome editing can lead to in-frame exon skipping
S Lalonde, OA Stone, S Lessard, A Lavertu, J Desjardins, M Beaudoin, M Rivas, DY Stainier, G Lettre, AS Lewin |
PloS one | 2017 |
Drugs in Development for Hepatitis B
A Dawood, SA Basit, M Jayaraj, RG Gish |
Drugs | 2017 |
Oligo- and dsDNA-mediated genome editing using a tetA dual selection system in Escherichia coli
YS Ryu, SP Chandran, K Kim, SK Lee, M Isalan |
PloS one | 2017 |
Human Germline Genome Editing
KE Ormond, DP Mortlock, DT Scholes, Y Bombard, LC Brody, WA Faucett, NA Garrison, L Hercher, R Isasi, A Middleton, K Musunuru, D Shriner, A Virani, CE Young |
The American Journal of Human Genetics | 2017 |
Self-cleaving ribozymes enable the production of guide RNAs from unlimited choices of promoters for CRISPR/Cas9 mediated genome editing
Y He, T Zhang, N Yang, M Xu, L Yan, L Wang, R Wang, Y Zhao |
Journal of Genetics and Genomics | 2017 |
Genome Editing
K Musunuru |
Journal of the American College of Cardiology | 2017 |
Producing defucosylated antibodies with enhanced in vitro antibody-dependent cellular cytotoxicity via FUT8 knockout CHO-S cells
H Zong, L Han, K Ding, J Wang, T Sun, X Zhang, C Cagliero, H Jiang, Y Xie, J Xu, B Zhang, J Zhu |
Engineering in Life Sciences | 2017 |
The next generation of antimicrobial peptides (AMPs) as molecular therapeutic tools for the treatment of diseases with social and economic impacts
NB da Cunha, NB Cobacho, JF Viana, LA Lima, KB Sampaio, SS Dohms, AC Ferreira, C de la Fuente-Núñez, FF Costa, OL Franco, SC Dias |
Drug Discovery Today | 2017 |
Recent Advances in Nucleic Acid Targeting Probes and Supramolecular Constructs Based on Pyrene-Modified Oligonucleotides.
Krasheninina OA, Novopashina DS, Apartsin EK, Venyaminova AG |
Molecules (Basel, Switzerland) | 2017 |
Emerging cellular and gene therapies for congenital anemias: Emerging Cellular and Gene Therapies for Congenital
LS Ludwig, RK Khajuria, VG Sankaran |
American Journal of Medical Genetics Part C Seminars in Medical Genetics | 2016 |
Genetics and Genomics of Coronary Artery Disease
M Pjanic, CL Miller, R Wirka, JB Kim, DM DiRenzo, T Quertermous |
Current Cardiology Reports | 2016 |
Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles
KR Yu, H Natanson, CE Dunbar |
Human Gene Therapy | 2016 |
Development and potential applications of CRISPR-Cas9 genome editing technology in sarcoma
T Liu, JK Shen, Z Li, E Choy, FJ Hornicek, Z Duan |
Cancer Letters | 2016 |
The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes
S Pierandrei, A Luchetti, M Sanchez, G Novelli, F Sangiuolo, M Lucarelli |
Molecular Therapy — Nucleic Acids | 2016 |
Harnessing the Prokaryotic Adaptive Immune System as a Eukaryotic Antiviral Defense
AA Price, A Grakoui, DS Weiss |
Trends in Microbiology | 2016 |
Genome editing in cardiovascular diseases
A Strong, K Musunuru |
Nature Reviews Cardiology | 2016 |
CT-Finder: A Web Service for CRISPR Optimal Target Prediction and Visualization
H Zhu, L Misel, M Graham, ML Robinson, C Liang |
Scientific Reports | 2016 |
Detection of nucleotide-specific CRISPR/Cas9 modified alleles using multiplex ligation detection
R KC, A Srivastava, JM Wilkowski, CE Richter, JA Shavit, DT Burke, SL Bielas |
Scientific Reports | 2016 |
A Broad Overview and Review of CRISPR-Cas Technology and Stem Cells
SN Waddington, R Privolizzi, R Karda, HC ONeill |
Current Stem Cell Reports | 2016 |
A 90-Day Feeding Study in Rats to Assess the Safety of Genetically Engineered Pork
G Xiao, SW Jiang, LL Qian, CB Cai, Q Wang, DZ Ma, B Li, S Xie, WT Cui, K Li, M Bader |
PloS one | 2016 |
Efficient, footprint-free human iPSC genome editing by consolidation of Cas9/CRISPR and piggyBac technologies
G Wang, L Yang, D Grishin, X Rios, LY Ye, Y Hu, K Li, D Zhang, GM Church, WT Pu |
Nature Protocols | 2016 |
Novel Thrombotic Function of a Human SNP in STXBP5 Revealed by CRISPR/Cas9 Gene Editing in MiceHighlights
QM Zhu, KA Ko, S Ture, MA Mastrangelo, MH Chen, AD Johnson, CJ ODonnell, CN Morrell, JM Miano, CJ Lowenstein |
Arteriosclerosis, thrombosis, and vascular biology | 2016 |
Cure for thalassemia major – from allogeneic hematopoietic stem cell transplantation to gene therapy
A Srivastava, RV Shaji |
Haematologica | 2016 |
Society for Pediatric Research 2015 Young Investigator Award: genetics of human hematopoiesis—what patients can teach us about blood cell production
A Wakabayashi, VG Sankaran |
Pediatric Research | 2015 |
Anemia: progress in molecular mechanisms and therapies
VG Sankaran, MJ Weiss |
Nature Medicine | 2015 |
Targeted Application of Human Genetic Variation Can Improve Red Blood Cell Production from Stem Cells
FC Giani, C Fiorini, A Wakabayashi, LS Ludwig, RM Salem, CD Jobaliya, SN Regan, JC Ulirsch, G Liang, O Steinberg-Shemer, MH Guo, T Esko, W Tong, C Brugnara, JN Hirschhorn, MJ Weiss, LI Zon, ST Chou, DL French, K Musunuru, VG Sankaran |
Cell Stem Cell | 2015 |
Progress and Prospects of Anti-HBV Gene Therapy Development
M Maepa, I Roelofse, A Ely, P Arbuthnot |
International journal of molecular sciences | 2015 |
Zebrafish as a disease model for studying human hepatocellular carcinoma
JW Lu |
World journal of gastroenterology : WJG | 2015 |
Genome engineering in cattle: recent technological advancements
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Chromosome Research | 2015 |
Toward stem cell-based phenotypic screens for neurodegenerative diseases
V Khurana, DF Tardiff, CY Chung, S Lindquist |
Nature Reviews Neurology | 2015 |
Chromatin organization in pluripotent cells: emerging approaches to study and disrupt function
CL Novo, PJ Rugg-Gunn |
Briefings in Functional Genomics | 2015 |
Plant artificial chromosome technology and its potential application in genetic engineering
W Yu, YY Yau, JA Birchler |
Plant Biotechnology Journal | 2015 |
2015 Guidelines for Establishing Genetically Modified Rat Models for Cardiovascular Research
MJ Flister, JW Prokop, J Lazar, M Shimoyama, M Dwinell, A Geurts |
Journal of Cardiovascular Translational Research | 2015 |
Neurology—the next 10 years
R Baron, DM Ferriero, GB Frisoni, C Bettegowda, ZL Gokaslan, JA Kessler, A Vezzani, SG Waxman, S Jarius, B Wildemann, M Weller |
Nature Reviews Neurology | 2015 |
Animal and human mucosal tissue models to study HIV biomedical interventions: can we predict success?
Dezzutti CS |
Journal of the International AIDS Society | 2015 |
Pulmonary surfactant: an immunological perspective
ZC Chroneos, Z Sever-Chroneos, VL Shepherd |
Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology | 2009 |