Αποτελέσματα Αναζήτησης
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,...
31 Μαΐ 2021 · Here, we review four platform technologies that have enabled the clinical translation of nucleic acid therapeutics: antisense oligonucleotides, ligand-modified small interfering RNA conjugates,...
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 cells in...
9 Νοε 2022 · In vivo gene immunotherapy approaches for engineering immune cells or modulating the tumor. Viral vectors and NPs may be engineered as targeted carriers of various payloads for durable or transient expression of therapeutic genes in tumor cells or immune cells.
31 Ιουλ 2019 · An example of in vivo gene therapy is the treatment of vision loss caused by loss-of-function variants in RPE65, which encodes an enzyme that converts all-trans-retinyl ester to 11-cis-retinol...
