Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome-Reference-Cited by-同舟云学术

Antisense oligonucleotide therapy in a humanized mouse model of MECP2 duplication syndrome

Published:2021-03-03 Issue:583 Volume:13 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Shao Yingyao¹²³^ORCID,Sztainberg Yehezkel¹²^ORCID,Wang Qi²⁴,Bajikar Sameer S.¹²^ORCID,Trostle Alexander J.²⁴,Wan Ying-Wooi¹²,Jafar-nejad Paymaan⁵,Rigo Frank⁵,Liu Zhandong²⁴^ORCID,Tang Jianrong²⁴^ORCID,Zoghbi Huda Y.¹²³⁴⁶^ORCID

Affiliation:

1. Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.

2. Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital, Houston, TX 77030, USA.

3. Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030, USA.

4. Department of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA.

5. Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

6. Howard Hughes Medical Institute, Baylor College of Medicine, Houston, TX 77030, USA.

Abstract

Antisense oligonucleotides are efficacious and safe in a humanized mouse model of MECP2 duplication syndrome.

Funder

National Institutes of Health

Howard Hughes Medical Institute

National Institute of General Medical Sciences

National Institute of Neurological Disorders and Stroke

National Institute of Child Health and Human Development

Rett Syndrome Research Trust

The 401 project

Cancer Prevention and Research Institute of Texas

Houston Endowment

Ting Tsung and Wei Fong Chao Family Foundation

Huffington Foundation

the Hamill Foundation

The Cockrell Foundation

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference42 articles.

1. The Role of MeCP2 in the Brain

2. MeCP2: only 100% will do

3. Submicroscopic duplication in Xq28 causes increased expression of the MECP2 gene in a boy with severe mental retardation and features of Rett syndrome

4. Duplication of the MECP2 Region Is a Frequent Cause of Severe Mental Retardation and Progressive Neurological Symptoms in Males

5. Increased MECP2 gene copy number as the result of genomic duplication in neurodevelopmentally delayed males

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