REVIEW
|
doi:10.20944/preprints202405.0274.v1
Subject:
Neuroscience And Neurology,
Medicine And Pharmacology
Keywords:
cell-penetrating peptides (CPP); antisense oligonucleotides (ASO); spinal muscular atrophy (SMA); delivery; phosphorodiamidate morpholino oligomers (PMO); DG9
Online: 6 May 2024 (09:54:35 CEST)
REVIEW
|
doi:10.20944/preprints202201.0265.v1
Subject:
Biology And Life Sciences,
Biochemistry And Molecular Biology
Keywords:
CRISPR; gene editing; Duchenne muscular dystrophy (DMD); exon skipping; NHEJ; dystrophin
Online: 18 January 2022 (17:30:37 CET)
REVIEW
|
doi:10.20944/preprints202311.0801.v1
Subject:
Medicine And Pharmacology,
Neuroscience And Neurology
Keywords:
Dysferlinopathy; limb-girdle muscular dystrophy recessive type 2 (LGMDR2); Miyoshi myopathy; distal myopathy with anterior tibial onset (DMAT); dysferlin; membrane resealing; genetic therapy; mini-dysferlin; exon skipping
Online: 13 November 2023 (10:41:28 CET)
REVIEW
|
doi:10.20944/preprints202309.1728.v1
Subject:
Medicine And Pharmacology,
Medicine And Pharmacology
Keywords:
Fibrodysplasia ossificans progressiva (FOP); heterotopic ossification; bone morphogenetic proteins (BMPs); ACVR1; antisense therapy
Online: 26 September 2023 (05:27:10 CEST)
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/preprints202301.0139.v1
Subject:
Medicine And Pharmacology,
Pediatrics, Perinatology And Child Health
Keywords:
eteplirsen; golodirsen; viltolarsen; casimersen; WVE-N531; SRP-5051; DS-5141B; NS-089/NCNP-02; SCAAV9.U7.ACCA; ATL1102
Online: 9 January 2023 (04:18:24 CET)
REVIEW
|
doi:10.20944/preprints201912.0385.v1
Subject:
Medicine And Pharmacology,
Cardiac And Cardiovascular Systems
Keywords:
dilated cardiomyopathy (DCM); hypertrophic cardiomyopathy (HCM); restrictive cardiomyopathy (RCM); arrhythmogenic right ventricular cardiomyopathy (ARVC); left ventricular non-compaction cardiomyopathy (LVNC); Duchenne muscular dystrophy; dystrophin; genome editing; CRISPR/Cas9; Cpf1 (Cas12a)
Online: 29 December 2019 (13:41:48 CET)
REVIEW
|
doi:10.20944/preprints201811.0018.v1
Subject:
Biology And Life Sciences,
Biochemistry And Molecular Biology
Keywords:
Duchenne muscular dystrophy (DMD); CRISPR/Cas9; exon skipping therapy; gene editing; human induced pluripotent stem cells (hiPSCs); immortalized patient muscle cells; mdx mice; humanized dystrophic mouse models; deltaE50-MD dog model
Online: 2 November 2018 (05:14:23 CET)
REVIEW
|
doi:10.20944/preprints202307.1409.v1
Subject:
Biology And Life Sciences,
Life Sciences
Keywords:
spinal and bulbar muscular atrophy; antisense therapy; oligonucleotide; splice switching; mRNA knockdown; androgen receptor; AR45
Online: 20 July 2023 (09:53:18 CEST)
REVIEW
|
doi:10.20944/preprints202003.0048.v1
Subject:
Biology And Life Sciences,
Biochemistry And Molecular Biology
Keywords:
Duchenne muscular dystrophy; CRISPR; animal models; in vivo testing; dystrophin; mutant generation
Online: 4 March 2020 (04:58:48 CET)
ARTICLE
|
doi:10.20944/preprints202303.0389.v1
Subject:
Computer Science And Mathematics,
Artificial Intelligence And Machine Learning
Keywords:
antisense oligonucleotides; exon skipping; machine learning; ensemble learning; personalized medicine; n-of-1 therapy, splice switching; genetic disease; splicing; RNA
Online: 22 March 2023 (03:24:48 CET)
ARTICLE
|
doi:10.20944/preprints202303.0167.v1
Subject:
Biology And Life Sciences,
Biology And Biotechnology
Keywords:
antisense oligonucleotides; exon skipping; machine learning; ensemble learning; personalized medicine; n-of-1 therapy; splice switching; genetic disease; splicing; RNA
Online: 9 March 2023 (04:43:55 CET)