Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder-Reference-Cited by-同舟云学术

Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder

Published:2020-03-01 Issue:3 Volume:143 Page:716-718
ISSN:0006-8950
Container-title:Brain
language:en
Short-container-title:

Author:

Benke Tim A¹²,Kind Peter C³

Affiliation:

1. Neuroscience Institute, Children’s Hospital Colorado, Aurora, CO, USA

2. University of Colorado School of Medicine, Aurora, CO, USA

3. Patrick Wild Centre, Edinburgh, UK

Abstract

This scientific commentary refers to ‘Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder’, by Gao etal. (doi:10.1093/brain/awaa028).

Funder

International Rett Syndrome Foundation

Rett Syndrome Research Trust

NIH

Children’s Hospital Colorado Foundation Ponzio Family Chair in Neurology Research

Loulou Foundation

Simons Foundation Autism Research Initiative

Medical Research Council

Publisher

Oxford University Press (OUP)

Subject

Neurology (clinical)

Link

http://academic.oup.com/brain/article-pdf/143/3/716/32963591/awaa055.pdf

Reference13 articles.

1. CDKL5 deficiency disorder: relationship between genotype, epilepsy, cortical visual impairment, and development;Demarest;Epilepsia,2019

2. Open-label use of highly purified CBD (Epidiolex(R)) in patients with CDKL5 deficiency disorder and Aicardi, Dup15q, and Doose syndromes;Devinsky;Epilepsy Behav,2018

3. Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder;Gao;Brain,2020

4. Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome;Garg;J Neurosci,2013

5. Reversal of neurological defects in a mouse model of Rett syndrome;Guy;Science,2007

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1. CDKL5 sculpts functional callosal connectivity to promote cognitive flexibility;Molecular Psychiatry;2023-02-03

2. Expression of a Secretable, Cell-Penetrating CDKL5 Protein Enhances the Efficacy of Gene Therapy for CDKL5 Deficiency Disorder;Neurotherapeutics;2022-09-15

3. Factors influencing the attainment of major motor milestones in CDKL5 deficiency disorder;European Journal of Human Genetics;2022-08-18

4. Electrophysiological biomarkers of brain function in CDKL5 deficiency disorder;Brain Communications;2022-07-04

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