Title: | Adeno-associated virus vector-based gene therapies for pediatric diseases |
Journal : | Pediatrics and neonatology |
Authors: | Muramatsu K ; Muramatsu SI |
Material Type: | Article |
Publisher: | Singapore, 30/09/2022 |
Languages: | English |
Keywords : | adenoassociated vectors ; adverse side effects ; Duchenne muscular dystrophy ; gene therapy ; hemophilia ; metabolic diseases ; pediatrics ; review article ; spinal muscular atrophy ; spinocerebellar ataxia ; Wilson disease ; X-linked myotubular myopathy |
Keywords: | AAVvector ; adeno-associated virus ; gene therapy ; pediatric disease |
Pubmed / DOI : | Pubmed : 36266189 / DOI : 10.1016/j.pedneo.2022.09.004 |
N° Profil MNM : | 2022101 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/36266189 |