Gene therapy has been an exciting development; once a theoretical dream, it is now, reality. The Archivist has been following the progress of this intervention and now seen patients who have received this 'miraculous' therapy. We know that spinal muscular atrophy (type 1) (SMA) is a ...
Early treatment is a lifeline for infants with SMA In the phase 3 SPR1NT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of spinal muscular atrophy (SMA); long-term safety follow-up of these patients must now be a key priority...
21. Aiuti A, Cattaneo F, Galimberti S, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009;360:447-458. 22. Cicalese MP, Ferrua F, Castagnaro L, et al. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due t...
Both tested the gene therapy in babies up to 6 months of age with type 1 SMA.Phase 1 START studyThe START study enrolled 15 babies who had already begun to experience disease symptoms. The children received ascending doses of the gene therapy and were monitored for two years, after which ...
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Mol Ther 2015; 23: 477–487. CAS PubMed Google Scholar Hua Y, Sahashi K, Rigo F, Hung G, Horev G, Bennett CF et al. Peripheral SMN restoration is ...
Our ultimate goal is to apply Sma I to gene therapy for this disease, because the mutant mitochondrial DNA (mtDNA) coexists with the wild-type mtDNA (heteroplasmy), and because only the mutant mtDNA, but not the wild-type mtDNA, is selectively restricted by the enzyme. For this purpose, ...
Learn about the only one-time gene therapy for SMA and see why treating early is so important. See safety and Full Prescribing Info. For US audiences only.
Modelling ASD in rodents: a platform for proof-of-principle studies The field of gene therapy is littered with examples of therapies which failed to translate from their preclinical promise. In many cases, blame can be attributed to the predictive validity of the animal model used, which is its...
gene therapy, and each vector serotype may have a distinctive safety profile26,27. Delandistrogene moxeparvovec uses the rAAVrh74 vector, a clade E AAV28, distinct from the AAV9 clade F vector used in some DMD clinical trials and an approved gene therapy for spinal muscular atrophy29,30...
A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published today in the New England Journal of Medicine. The study was conducted by Researchers from...