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Dystrophin gene editing stability is dependent on dystrophin levels in skeletal but not cardiac muscles
Bengtsson NE, Tasfaout H, Hauschka SD, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
DUX4 Transcript Knockdown with Antisense 2'-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy
Lim KRQ, Bittel A, Maruyama R, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice
Besse A, Astord S, Marais T, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid alpha-Glucosidase
Cagin U, Puzzo F, Gomez MJ, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
AMPK Complex Activation Promotes Sarcolemmal Repair in Dysferlinopathy
Ono H, Suzuki N, Kanno SI, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing
Min YL, Chemello F, Li H, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2020, 28, 9, p 2044
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Verdera HC, Kuranda K, Mingozzi F
Molecular therapy : the journal of the American Society of Gene Therapy, 2020
biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for alpha-Dystroglycanopathies
Gumlaw N, Sevigny LM, Zhao H, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019
AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy
Dupont JB, Guo J, Renaud-Gabardos E, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
Nance ME, Shi R, Hakim CH, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 9, p. 1568-1585
Gene Correction of LGMD2A Patient-Specific iPSCs for the Development of Targeted Autologous Cell Therapy
Selvaraj S, Dhoke NR, Kiley J, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019
Effect of Cryopreservation on Autologous Chimeric Antigen Receptor T Cell Characteristics
Panch SR, Srivastava SK, Elavia N, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 7, p 1275
Fresh versus Frozen: Effects of Cryopreservation on CAR T Cells
Hanley PJ
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 7, p 1213
Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene
Echigoya Y, Lim KRQ, Melo D, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019
Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice.
Lo Scrudato M, Poulard K, Sourd C, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019
Development of Novel Micro-dystrophins with Enhanced Functionality
Ramos JN, Hollinger K, Bengtsson NE, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 3, p 623
Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy
Davies KE, Davies KE, Guiraud S
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 3, p 486
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Duan D
Molecular therapy : the journal of the American Society of Gene Therapy, 2018, 26, 10, p 2337
CRISPR-Induced Deletion with SaCas9 Restores Dystrophin Expression in Dystrophic Models In Vitro and In Vivo
Duchene BL, Duchene BL, Cherif K, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018
Therapeutic Benefit of Autophagy Modulation in Pompe Disease
Lim JA, Sun B, Puertollano R, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018
Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice
Tasfaout H, Lionello VM, Kretz C, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with RPE65 Leber Congenital Amaurosis
Le Meur G, Lebranchu P, Billaud F, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018, 26, 1, p 256
Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease
Koeberl DD, Koeberl DD, Case LE, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018, 26, 9, p 2304
Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit
Sreetama SC, Chandra G, Van Der Meulen JH, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2018, 26, 9, p 2231
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Vidal P, Pagliarani S, Colella P, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2017
