Literature DB >> 35585789

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.

Beverly L Davidson1, Guangping Gao2, Elizabeth Berry-Kravis3, Allison M Bradbury4, Carsten Bönnemann5, Joseph D Buxbaum6, Gavin R Corcoran7, Steven J Gray8, Heather Gray-Edwards9, Robin J Kleiman10, Adam J Shaywitz11, Dan Wang12, Huda Y Zoghbi13, Terence R Flotte14, Sitra Tauscher-Wisniewski15, Cynthia J Tifft16, Mustafa Sahin17.   

Abstract

We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.
Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved.

Entities:  

Keywords:  gene therapy; neurodevelopment; psychiatric disorders

Mesh：

Year:  2022        PMID： 35585789      PMCID： PMC9263284          DOI： 10.1016/j.ymthe.2022.05.014

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


  112 in total

Review 1.  Gene therapy for neurological disorders: progress and prospects.

Authors:  Benjamin E Deverman; Bernard M Ravina; Krystof S Bankiewicz; Steven M Paul; Dinah W Y Sah
Journal:  Nat Rev Drug Discov       Date:  2018-08-10       Impact factor: 84.694

Review 2.  Recent endeavors in MECP2 gene transfer for gene therapy of Rett syndrome.

Authors:  Sarah E Sinnett; Steven J Gray
Journal:  Discov Med       Date:  2017-10       Impact factor: 2.970

3.  Ube3a reinstatement identifies distinct developmental windows in a murine Angelman syndrome model.

Authors:  Sara Silva-Santos; Geeske M van Woerden; Caroline F Bruinsma; Edwin Mientjes; Mehrnoush Aghadavoud Jolfaei; Ben Distel; Steven A Kushner; Ype Elgersma
Journal:  J Clin Invest       Date:  2015-04-13       Impact factor: 14.808

Review 4.  10 Years of GWAS Discovery: Biology, Function, and Translation.

Authors:  Peter M Visscher; Naomi R Wray; Qian Zhang; Pamela Sklar; Mark I McCarthy; Matthew A Brown; Jian Yang
Journal:  Am J Hum Genet       Date:  2017-07-06       Impact factor: 11.025

5.  Meta-analysis and multidisciplinary consensus statement: exome sequencing is a first-tier clinical diagnostic test for individuals with neurodevelopmental disorders.

Authors:  Siddharth Srivastava; Jamie A Love-Nichols; Kira A Dies; David H Ledbetter; Christa L Martin; Wendy K Chung; Helen V Firth; Thomas Frazier; Robin L Hansen; Lisa Prock; Han Brunner; Ny Hoang; Stephen W Scherer; Mustafa Sahin; David T Miller
Journal:  Genet Med       Date:  2019-06-11       Impact factor: 8.822

6.  A systematic capsid evolution approach performed in vivo for the design of AAV vectors with tailored properties and tropism.

Authors:  Marcus Davidsson; Gang Wang; Patrick Aldrin-Kirk; Tiago Cardoso; Sara Nolbrant; Morgan Hartnor; Janitha Mudannayake; Malin Parmar; Tomas Björklund
Journal:  Proc Natl Acad Sci U S A       Date:  2019-12-09       Impact factor: 11.205

7.  Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction.

Authors:  Manish Muhuri; Wei Zhan; Yukiko Maeda; Jia Li; Anoushka Lotun; Jennifer Chen; Katelyn Sylvia; Ishani Dasgupta; Motahareh Arjomandnejad; Thomas Nixon; Allison M Keeler; Sangeetha Manokaran; Ran He; Qin Su; Phillip W L Tai; Guangping Gao
Journal:  Front Immunol       Date:  2021-04-28       Impact factor: 8.786

8.  A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system.

Authors:  Oleksandr Kondratov; Liudmyla Kondratova; Ronald J Mandel; Kirsten Coleman; Michael A Savage; Heather L Gray-Edwards; Timothy J Ness; Edgar Rodriguez-Lebron; Robert D Bell; Joseph Rabinowitz; Paul D Gamlin; Sergei Zolotukhin
Journal:  Mol Ther       Date:  2021-07-21       Impact factor: 12.910

9.  Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Authors:  Brittney L Gurda; Adrien De Guilhem De Lataillade; Peter Bell; Yanqing Zhu; Hongwei Yu; Ping Wang; Jessica Bagel; Charles H Vite; Tracey Sikora; Christian Hinderer; Roberto Calcedo; Alexander D Yox; Richard A Steet; Therese Ruane; Patricia O'Donnell; Guangping Gao; James M Wilson; Margret Casal; Katherine P Ponder; Mark E Haskins
Journal:  Mol Ther       Date:  2015-10-08       Impact factor: 11.454

Review 10.  Heritability of autism spectrum disorders: a meta-analysis of twin studies.

Authors:  Beata Tick; Patrick Bolton; Francesca Happé; Michael Rutter; Frühling Rijsdijk
Journal:  J Child Psychol Psychiatry       Date:  2015-12-27       Impact factor: 8.982
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  1 in total

Review 1.  GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy.

Authors:  Maya Chopra; Meera E Modi; Kira A Dies; Nancy L Chamberlin; Elizabeth D Buttermore; Stephanie Jo Brewster; Lisa Prock; Mustafa Sahin
Journal:  Mol Ther Methods Clin Dev       Date:  2022-08-29       Impact factor: 5.849
  1 in total

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