Literature DB >> 30946822

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.

Eloise Hudry, Luk H Vandenberghe.   

Abstract

Year:  2019        PMID： 30946822     DOI： 10.1016/j.neuron.2019.03.020

Source DB:  PubMed          Journal:  Neuron        ISSN： 0896-6273            Impact factor:   17.173


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  22 in total

1.  Treatment of a Mouse Model of ALS by In Vivo Base Editing.

Authors:  Colin K W Lim; Michael Gapinske; Alexandra K Brooks; Wendy S Woods; Jackson E Powell; M Alejandra Zeballos C; Jackson Winter; Pablo Perez-Pinera; Thomas Gaj
Journal:  Mol Ther       Date:  2020-01-14       Impact factor: 11.454

2.  Splicing repression is a major function of TDP-43 in motor neurons.

Authors:  Aneesh Donde; Mingkuan Sun; Jonathan P Ling; Kerstin E Braunstein; Bo Pang; Xinrui Wen; Xueying Cheng; Liam Chen; Philip C Wong
Journal:  Acta Neuropathol       Date:  2019-07-22       Impact factor: 17.088

3.  Adeno-associated virus (AAV) 9-mediated gene delivery of Nurr1 and Foxa2 ameliorates symptoms and pathologies of Alzheimer disease model mice by suppressing neuro-inflammation and glial pathology.

Authors:  Yunseon Yang; Min-Jong Seok; Ye Eun Kim; Yunjung Choi; Jae-Jin Song; Yanuar Alan Sulistio; Seong-Hoon Kim; Mi-Yoon Chang; Soo-Jin Oh; Min-Ho Nam; Yun Kyung Kim; Tae-Gyun Kim; Heh-In Im; Seong-Ho Koh; Sang-Hun Lee
Journal:  Mol Psychiatry       Date:  2022-07-29       Impact factor: 13.437

Review 4.  Neurodevelopmental copy-number variants: A roadmap to improving outcomes by uniting patient advocates, researchers, and clinicians for collective impact.

Authors: 
Journal:  Am J Hum Genet       Date:  2022-08-04       Impact factor: 11.043

5.  Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.

Authors:  Jonathan B Rosenberg; Alvin Chen; Stephen M Kaminsky; Ronald G Crystal; Dolan Sondhi
Journal:  Expert Opin Orphan Drugs       Date:  2019-11-27       Impact factor: 0.694

6.  A Single Intravenous Injection of AAV-PHP.B-hNDUFS4 Ameliorates the Phenotype of Ndufs4 -/- Mice.

Authors:  Pedro Silva-Pinheiro; Raffaele Cerutti; Marta Luna-Sanchez; Massimo Zeviani; Carlo Viscomi
Journal:  Mol Ther Methods Clin Dev       Date:  2020-05-04       Impact factor: 6.698

7.  Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.

Authors:  Giulia Massaro; Michael P Hughes; Sammie M Whaler; Kerri-Lee Wallom; David A Priestman; Frances M Platt; Simon N Waddington; Ahad A Rahim
Journal:  Hum Mol Genet       Date:  2020-07-29       Impact factor: 6.150

8.  Cell-Mediated Immunity to NAGLU Transgene Following Intracerebral Gene Therapy in Children With Mucopolysaccharidosis Type IIIB Syndrome.

Authors:  Marie-Lise Gougeon; Béatrice Poirier-Beaudouin; Jérome Ausseil; Michel Zérah; Cécile Artaud; Jean-Michel Heard; Kumaran Deiva; Marc Tardieu
Journal:  Front Immunol       Date:  2021-05-10       Impact factor: 7.561

Review 9.  Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

Authors:  Wei Zhan; Manish Muhuri; Phillip W L Tai; Guangping Gao
Journal:  Front Immunol       Date:  2021-05-11       Impact factor: 7.561

10.  Region and Cell Type Distribution of TCF4 in the Postnatal Mouse Brain.

Authors:  Hyojin Kim; Noah C Berens; Nicole E Ochandarena; Benjamin D Philpot
Journal:  Front Neuroanat       Date:  2020-07-17       Impact factor: 3.856
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