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Nature Chemical Biology, ISSN 1552-4450, 07/2015, Volume 11, Issue 7, pp. 511 - 517
Journal Article
Journal of Medicinal Chemistry, ISSN 0022-2623, 12/2018, Volume 61, Issue 24, pp. 11021 - 11036
Spinal muscular atrophy (SMA), a rare neuromuscular disorder, is the leading genetic cause of death in infants and toddlers. SMA is caused by the deletion or a... 
CHEMISTRY, MEDICINAL | MODIFIERS | IDENTIFICATION | SINGLE NUCLEOTIDE
Journal Article
Human Gene Therapy Clinical Development, ISSN 2324-8637, 12/2018, Volume 29, Issue 4, pp. 188 - 197
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco,... 
retinitis pigmentosa | gene therapy | XLRP | AAV | RPGR | MEDICINE, RESEARCH & EXPERIMENTAL | ISOFORMS | EFFICACY | RPGR-ORF15 | STABILITY | ROD | BIOTECHNOLOGY & APPLIED MICROBIOLOGY | DISEASE | GENE-THERAPY | LINKED RETINITIS-PIGMENTOSA | GLUTAMYLATION | Index Medicus
Journal Article
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