Title: | Antisense oligonucleotides and other genetic therapies made simple. |
Journal : | Practical Neurology, 18, 2 |
Authors: | Rossor AM, Author ; Reilly MM ; Sleigh JN |
Material Type: | Article |
Publication Date: | 2018 |
Size: | p 126 |
Languages: | English |
Keywords : | adenoassociated vectors ; antisense oligonucleotides ; CRISPR/Cas9 ; Duchenne muscular dystrophy ; eteplirsen ; exon skipping ; familial amyloid neuropathy ; gene therapy ; genome editing ; interfering RNA ; medical treatment ; nusinersen ; patisiran ; reviews ; SMN1-related proximal spinal muscular atrophy ; splicing ; therapeutic prospects ; viral vectors |
Keywords: | Rna interference;antisense oligonucleotides;genome editing;neurogenetics;viral gene therapy; |
Pubmed / DOI : | DOI : 10.1136/practneurol-2017-001764 / Pubmed : 29455156 |
N° Profil MNM : | 2018031 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/29455156 |