Molecular therapy for polyQ disorders: from bench to clinical trials-Reference-Cited by-同舟云学术

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Molecular therapy for polyQ disorders: from bench to clinical trials

Published:2024-09 Issue:9 Volume:30 Page:804-808
ISSN:1471-4914
Container-title:Trends in Molecular Medicine
language:en
Short-container-title:Trends in Molecular Medicine

Author:

de Sousa-Lourenço João^ORCID,Silva Ana C.^ORCID,Pereira de Almeida Luís^ORCID,Nobre Rui J.^ORCID

Publisher

Elsevier BV

Reference14 articles.

1. Polyglutamine repeats in neurodegenerative diseases;Lieberman;Annu. Rev. Pathol. Mech. Dis.,2019

2. Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders;Silva;Brain,2020

3. Principles for targeting RNA with drug-like small molecules;Warner;Nat. Rev. Drug Discov.,2018

4. Engineering adeno-associated virus vectors for gene therapy;Li;Nat. Rev. Genet.,2020

5. Insulin-like growth factor 2 (IGF2) protects against Huntington’s disease through the extracellular disposal of protein aggregates;García-Huerta;Acta Neuropathol.,2020

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