Current clinical applications of in vivo gene therapy with AAVs

JR Mendell, SA Al-Zaidy, LR Rodino-Klapac… - Molecular Therapy, 2021 - cell.com
Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

L Kang, S **, J Wang, Z Lv, C ** novel therapeutic strategies for Alzheimer's disease
J Cao, J Hou, J **, D Cai - Molecular neurodegeneration, 2018 - Springer
Abstract Alzheimer's Disease (AD), the most prevalent neurodegenerative disease of aging,
affects one in eight older Americans. Nearly all drug treatments tested for AD today have …

Design of virus-based nanomaterials for medicine, biotechnology, and energy

AM Wen, NF Steinmetz - Chemical Society Reviews, 2016 - pubs.rsc.org
This review provides an overview of recent developments in “chemical virology.” Viruses, as
materials, provide unique nanoscale scaffolds that have relevance in chemical biology and …

Adeno-associated virus-based gene therapy for CNS diseases

M Hocquemiller, L Giersch, M Audrain… - Human gene …, 2016 - liebertpub.com
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Nucleus basalis of Meynert revisited: anatomy, history and differential involvement in Alzheimer's and Parkinson's disease

AKL Liu, RCC Chang, RKB Pearce… - Acta neuropathologica, 2015 - Springer
It has been well established that neuronal loss within the cholinergic nucleus basalis of
Meynert (nbM) correlates with cognitive decline in dementing disorders such as Alzheimer's …

Nerve growth factor gene therapy: activation of neuronal responses in Alzheimer disease

MH Tuszynski, JH Yang, D Barba, U Hoi-Sang… - JAMA …, 2015 - jamanetwork.com
Importance Alzheimer disease (AD) is the most common neurodegenerative disorder and
lacks effective disease-modifying therapies. In 2001, we initiated a clinical trial of nerve …