Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1-Reference-Cited by-同舟云学术

Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1

Published:2019-12 Issue: Volume:15 Page:18-26
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Prabhakar Shilpa,Cheah Pike See,Zhang Xuan,Zinter Max,Gianatasio Maria,Hudry Eloise,Bronson Roderick T.,Kwiatkowski David J.,Stemmer-Rachamimov Anat,Maguire Casey A.,Sena-Esteves Miguel,Tannous Bakhos A.,Breakefield Xandra O.

Funder

U.S. Department of Defense

National Institutes of Health

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference33 articles.

1. Tuberous sclerosis complex;Henske;Nat. Rev. Dis. Primers,2016

2. A complex interplay between Akt, TSC2 and the two mTOR complexes;Huang;Biochem. Soc. Trans.,2009

3. Evolving neurobiology of tuberous sclerosis complex;Crino;Acta Neuropathol.,2013

4. Tuberous Sclerosis Complex: Genes, Clinical Features, and Therapeutics;Kwiatkowski,2010

5. Tuberous sclerosis complex and neurofibromatosis type 1: the two most common neurocutaneous diseases;Kandt;Neurol. Clin.,2003

Cited by 19 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Tuberous Sclerosis Complex and the kidneys: what nephrologists need to know;Brazilian Journal of Nephrology;2024-09

2. Complexo da Esclerose Tuberosa e rins: o que os nefrologistas devem saber;Brazilian Journal of Nephrology;2024-09

3. HIV-1 mRNA knockdown with CRISPR/CAS9 enhances neurocognitive function;Journal of NeuroVirology;2024-02

4. HIV-1 mRNA Knockdown with CRISPR/Cas9 Enhances Neurocognitive Function;2023-10-16

5. AAV-based in vivo gene therapy for neurological disorders;Nature Reviews Drug Discovery;2023-09-01