JCI - Citations to Gene therapy for severe combined immunodeficiency: are we there yet?

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Gene therapy for severe combined immunodeficiency: are we there yet?

Marina Cavazzana-Calvo, Alain Fischer

Marina Cavazzana-Calvo, Alain Fischer

Published June 1, 2007
Citation Information: J Clin Invest. 2007;117(6):1456-1465. https://doi.org/10.1172/JCI30953.

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Citations to this article (55)

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Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID L Garcia-Perez, M van Eggermond, L van Roon, SA Vloemans, M Cordes, A Schambach, M Rothe, D Berghuis, C Lagresle-Peyrou, M Cavazzana, F Zhang, AJ Thrasher, D Salvatori, P Meij, A Villa, JJ Dongen, JJ Zwaginga, M van der Burg, HB Gaspar, A Lankester, FJ Staal, K Pike-Overzet	Molecular Therapy — Methods & Clinical Development	2020
Darwinian selection within an individual or somatic selection: facts and models RA Veitia	Journal of Molecular Cell Biology	2019
ADA Deficiency: Evaluation of the Clinical and Laboratory Features and the Outcome D Cagdas, PG Cetinkaya, B Karaatmaca, S Esenboga, C Tan, T Yılmaz, E Gümüş, S Barış, B Kuşkonmaz, TT Ozgur, P Bali, I Santisteban, D Orhan, A Yüce, D Cetinkaya, K Boztug, M Hershfield, O Sanal, İ Tezcan	Journal of Clinical Immunology	2018
Advances in gene therapy for muscular dystrophies H Abdul-Razak, A Malerba, G Dickson	F1000Research	2016
Gene therapy for metabolic diseases RJ Chandler, CP Venditti	Translational Science of Rare Diseases	2016
Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy Murtaza S Nagree, Lucía López-Vásquez, Jeffrey A Medin	World journal of stem cells	2015
Insights into the nuclear export of murine leukemia virus intron-containing RNA L Pessel-Vivares, L Houzet, S Lainé, M Mougel	RNA biology	2015
Characterization of Equine Infectious Anemia Virus Integration in the Horse Genome Q Liu, XF Wang, J Ma, XJ He, XJ Wang, JH Zhou	Viruses	2015
Gene therapy for ocular diseases meditated by ultrasound and microbubbles (Review) C Wan, F Li, H Li	Molecular medicine reports	2015
Assessing risk/benefit for trials using preclinical evidence: a proposal J Kimmelman, V Henderson	Journal of medical ethics	2015
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No evidence of clonal dominance after transplant of HOXB4-expanded cord blood cells in a nonhuman primate model KL Watts, BC Beard, BL Wood, GD Trobridge, RK Humphries, AB Adams, V Nelson, HP Kiem	Experimental Hematology	2014
MLV requires Tap/NXF1-dependent pathway to export its unspliced RNA to the cytoplasm and to express both spliced and unspliced RNAs L Pessel-Vivares, M Ferrer, S Lainé, M Mougel	Retrovirology	2014
Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches RJ Fairclough, MJ Wood, KE Davies	Nature Reviews Genetics	2013
The era of genomic medicine K Davies	Clinical Medicine	2013
Gene therapy model of X-linked severe combined immunodeficiency using a modified foamy virus vector S Horino, T Uchiyama, T So, H Nagashima, SL Sun, M Sato, A Asao, Y Haji, Y Sasahara, F Candotti, S Tsuchiya, S Kure, K Sugamura, N Ishii	PloS one	2013
Gene therapy for cardiovascular disease mediated by ultrasound and microbubbles ZY Chen, Y Lin, F Yang, L Jiang, S Ge	Cardiovascular ultrasound	2013
Development of safer gene delivery systems to minimize the risk of insertional mutagenesis-related malignancies: a critical issue for the field of gene therapy G Romano	ISRN Oncology	2012
Novel Cell and Gene Therapies for HIV JA Hoxie, CH June		2012
Nanoscale Particles for Lung Delivery of siRNA Nascimento TL, Hillaireau H, Fattal E	Journal of drug delivery science and technology	2012
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AAV and Compacted DNA Nanoparticles for the Treatment of Retinal Disorders: Challenges and Future Prospects Z Han, SM Conley, MI Naash	Investigative ophthalmology & visual science	2011
Adipocytes as a vehicle for ex vivo gene therapy: Novel replacement therapy for diabetes and other metabolic diseases M Kuroda, H Bujo, M Aso, Y Saito	Journal of Diabetes Investigation	2011
Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency JN Silver, Melissa, T Conlon, P Cruz, AJ Wright, A Srivastava, TR Flotte	Human Gene Therapy	2011
Lentiviral vectors for immune cells targeting S Froelich, A Tai, P Wang	Immunopharmacology and Immunotoxicology	2010
A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells S Zhou, D Mody, SS DeRavin, J Hauer, T Lu, Z Ma, SH Abina, JT Gray, MR Greene, M Cavazzana-Calvo, HL Malech, BP Sorrentino	Blood	2010
B cell-specific lentiviral gene therapy leads to sustained B-cell functional recovery in a murine model of X-linked agammaglobulinemia HM Kerns, BY Ryu, BV Stirling, BD Sather, A Astrakhan, S Humblet-Baron, D Liggitt, DJ Rawlings	Blood	2010
The Biology of Allogeneic Hematopoietic Cell Resistance JA Shizuru, D Bhattacharya, M Cavazzana-Calvo	Biology of Blood and Marrow Transplantation	2010
Retroviral Integration Site Selection S Desfarges, A Ciuffi	Viruses	2010
Novel Viral Vector Systems for Gene Therapy D Stone	Viruses	2010
Induction of Pluripotent Stem Cells from Human Third Molar Mesenchymal Stromal Cells*♦ Y Oda, Y Yoshimura, H Ohnishi, M Tadokoro, Y Katsube, M Sasao, Y Kubo, K Hattori, S Saito, K Horimoto, S Yuba, H Ohgushi	The Journal of biological chemistry	2010
Chromatin tethering and retroviral integration: Recent discoveries and parallels with DNA viruses AM Meehan, EM Poeschla	Biochimica et Biophysica Acta (BBA) - Gene Regulatory Mechanisms	2010
Biomolecular Structure and Modeling: Problem and Application Perspective Schlick T		2010
Chronic Granulomatous Disease SM Holland	Clinical Reviews in Allergy & Immunology	2009
Finding the needle in the hay stack: Hematopoietic stem cells in Fanconi anemia LU Müller, DA Williams	Mutation Research/Fundamental and Molecular Mechanisms of Mutagenesis	2009
Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression E Kimura, S Li, P Gregorevic, BM Fall, JS Chamberlain	Molecular Therapy	2009
Stimulation of homology-directed gene targeting at an endogenous human locus by a nicking endonuclease GP van Nierop, AA de Vries, M Holkers, KR Vrijsen, MA Gonçalves	Nucleic Acids Research	2009
Alphaviruses in Gene Therapy K Lundstrom	Viruses	2009
Polyethylenimine-mediated gene delivery to the lung and therapeutic applications Di Gioia S, Conese M	Drug design, development and therapy	2009
High Incidence of Leukemia after Stem-Cell Gene Therapy in Large Animals with a HOXB4-expressing Vector Xiao-Bing Zhang, Brian C. Beard, Grant D. Trobridge, Brent L. Wood, George E. Sale, Reeteka Sud, R. Keith Humphries, Hans-Peter Kiem	Journal of Clinical Investigation	2008
Evolution of highly polymorphic T cell populations in siblings with the Wiskott-Aldrich Syndrome MI Lutskiy, JY Park, SK Remold, E Remold-O'Donnell	PloS one	2008
Cell type differences in activity of the Streptomyces bacteriophage phiC31 integrase C Maucksch, MK Aneja, E Hennen, A Bohla, F Hoffmann, M Elfinger, J Rosenecker, C Rudolph	Nucleic Acids Research	2008
Mutations in CHD7 in patients with CHARGE syndrome cause T-B + natural killer cell + severe combined immune deficiency and may cause Omenn-like syndrome AR Gennery, MA Slatter, J Rice, LH Hoefsloot, D Barge, A McLean-Tooke, T Montgomery, JA Goodship, AD Burt, TJ Flood, M Abinun, AJ Cant, D Johnson	Clinical & Experimental Immunology	2008
Hepatocyte Growth Factor Enhances Engraftment and Function of Nonhuman Primate Islets NM Fiaschi-Taesch, DM Berman, BM Sicari, KK Takane, A Garcia-Ocana, C Ricordi, NS Kenyon, AF Stewart	Diabetes	2008
Maintaining the silence: reflections on long-term RNAi K Raemdonck, RE Vandenbroucke, J Demeester, NN Sanders, SC Smedt	Drug Discovery Today	2008
Correction of glycogenosis type 2 by muscle-specific lentiviral vector E Richard, G Douillard-Guilloux, L Batista, C Caillaud	In vitro cellular & developmental biology. Animal	2008
Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options SS Ravin, HL Malech	Immunologic Research	2008
Transporter Molecules influence the Gene Expression in HeLa Cells Waldeck W, Pipkorn R, Korn B, Mueller G, Schick M, Tóth K, Wiessler M, Didinger B, Braun K	International journal of medical sciences	2008
Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy Alessandro Aiuti, Barbara Cassani, Grazia Andolfi, Massimiliano Mirolo, Luca Biasco, Alessandra Recchia, Fabrizia Urbinati, Cristina Valacca, Samantha Scaramuzza, Memet Aker, Shimon Slavin, Matteo Cazzola, Daniela Sartori, Alessandro Ambrosi, Clelia Di Serio, Maria Grazia Roncarolo, Fulvio Mavilio, and Claudio Bordignon.	Journal of Clinical Investigation	2007
Therapeutic application of RNAi: Is mRNA targeting finally ready for prime-time? Dirk Grimm and Mark A. Kay	Journal of Clinical Investigation	2007
Cellular genetic tools to control oncolytic adenoviruses for virotherapy of cancer DM Nettelbeck	Journal of Molecular Medicine	2007
Characterization of a natural heterodimer between MLV genomic RNA and the SD' retroelement generated by alternative splicing S Maurel, L Houzet, EL Garcia, A Telesnitsky, M Mougel	RNA (New York, N.Y.)	2007

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