REVIEW
|
doi:10.20944/preprints202308.1964.v1
Subject:
Medicine And Pharmacology,
Neuroscience And Neurology
Keywords:
Antisense Oligonucleotides; Cell Penetrating Peptides; Delivery; DG9 peptide; Phosphorodi-amidate morpholino oligomers (PMO); Pip; R6G
Online: 29 August 2023 (09:48:58 CEST)
REVIEW
|
doi:10.20944/preprints202406.2068.v1
Subject:
Biology And Life Sciences,
Neuroscience And Neurology
Keywords:
spinal muscular atrophy (SMA); survival of motor neuron 1 (SMN1); SMN2; SMN protein; antisense oligonucleotide (ASO); nusinersen; gene therapy; onasemnogene; risdiplam; small molecule; combination therapy
Online: 29 June 2024 (06:33:01 CEST)