Αποτελέσματα Αναζήτησης
1 Σεπ 2023 · The gene therapy, which used a recombinant adeno-associated virus serotype 2 (rAAV2) vector to deliver a functional ASPA gene, was safe and associated with modest phenotypic improvement and a...
- The once and future gene therapy | Nature Communications
For example, the success of in vivo AAV gene transfer to the...
- Four Success Stories in Gene Therapy - Nature
Luxturna was the first FDA-approved in vivo gene therapy,...
- The once and future gene therapy | Nature Communications
1 Νοε 2021 · One new arrival to the gene therapy scene is being watched particularly closely: in vivo gene editing using a system called CRISPR, which has become one of the most promising gene...
The review article presents a broad overview of the field of therapy by in vivo gene transfer, which is based on direct administration of a gene-therapy vector to the body rather than transplant of gene-corrected cells.
16 Νοε 2020 · For example, the success of in vivo AAV gene transfer to the human retina and central nervous system by Luxturna and Zolgensma for Leber’s congenital amaurosis and spinal muscular atrophy ...
3 Φεβ 2021 · We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's disease, Canavan disease, aromatic l-amino acid decarboxylase [AADC] deficiency, and giant axonal ...
26 Οκτ 2021 · Luxturna was the first FDA-approved in vivo gene therapy, which is delivered to target cells inside the body (previously approved ex vivo therapies deliver the genetic material to target...
3 Φεβ 2021 · The review article presents a broad overview of the field of therapy by in vivo gene transfer, which is based on direct administration of a gene-therapy vector to the body rather than transplant of gene-corrected cells.
