A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice-Reference-Cited by-同舟云学术

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A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice

Published:2022-07-01 Issue:13 Volume:132 Page:
ISSN:1558-8238
Container-title:Journal of Clinical Investigation
language:en
Short-container-title:

Author:

Stavrou Marina^ORCID,Kagiava Alexia^ORCID,Choudury Sarah G.,Jennings Matthew J.,Wallace Lindsay M.,Fowler Allison M.,Heslegrave Amanda,Richter Jan^ORCID,Tryfonos Christina,Christodoulou Christina^ORCID,Zetterberg Henrik^ORCID,Horvath Rita^ORCID,Harper Scott Q.,Kleopa Kleopas A.^ORCID

Funder

CMT Research Foundation

UK Dementia Research Institute

Wellcome Trust

Medical Research Council

Addenbrookes Charitable Trust

Evelyn Trust

Stoneygate Trust

Lily Foundation

MRC strategic award

Medical Research Council (MRC) (UK) DiMeN

Cambridge DTPs

NIHR Cambridge Biomedical Research Centre

Publisher

American Society for Clinical Investigation

Subject

General Medicine

Link

https://www.jci.org/articles/view/159814/files/pdf

Reference113 articles.

1. Genetic and clinical aspects of Charcot-Marie-Tooth’s disease;Skre;Clin Genet,1974

2. Epidemiology of peripheral neuropathy.

3. DNA duplication associated with Charcot-Marie-Tooth disease type 1A

4. Peripheral myelin protein–22 gene maps in the duplication in chromosome 17p11.2 associated with Charcot–Marie–Tooth 1A

5. The gene for the peripheral myelin protein PMP–22 is a candidate for Charcot–Marie–Tooth disease type 1A

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1. Current Treatment Methods for Charcot–Marie–Tooth Diseases;Biomolecules;2024-09-09

2. Navigating the Landscape of CMT1B: Understanding Genetic Pathways, Disease Models, and Potential Therapeutic Approaches;International Journal of Molecular Sciences;2024-08-26

3. Gene therapies for CMT neuropathies: from the bench to the clinic;Current Opinion in Neurology;2024-06-14

4. Lumbar Intrathecal Injection in Adult and Neonatal Mice;Current Protocols;2024-06

5. PMP22 duplication dysregulates lipid homeostasis and plasma membrane organization in developing human Schwann cells;Brain;2024-05-14

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