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 …

Recent advancements in liposomes targeting strategies to cross blood-brain barrier (BBB) for the treatment of Alzheimer's disease

M Agrawal, DK Tripathi, S Saraf, S Saraf… - Journal of controlled …, 2017 - Elsevier
In this modern era, with the help of various advanced technologies, medical science has
overcome most of the health-related issues successfully. Though, some diseases still remain …

[HTML][HTML] Gene therapy for neurodegenerative diseases

V Sudhakar, RM Richardson - Neurotherapeutics, 2019 - Elsevier
Gene therapy has the potential to provide therapeutic benefit to millions of people with
neurodegenerative diseases through several means, including direct correction of …

Adeno-associated viral vector (serotype 2)–nerve growth factor for patients with alzheimer disease: a randomized clinical trial

MS Rafii, MH Tuszynski, RG Thomas, D Barba… - JAMA …, 2018 - jamanetwork.com
Importance Nerve growth factor (NGF) is an endogenous neurotrophic factor that prevents
the death and augments the functional state of cholinergic neurons of the basal forebrain, a …

Neurotrophic factors in Alzheimer's and Parkinson's disease brain

GJ Siegel, NB Chauhan - Brain Research Reviews, 2000 - Elsevier
The biomedical literature on the subject of neurotrophic growth factors has expanded
prodigiously. This essay reviews neurotrophic factors (NTF) and their receptors in …

The biology of neurotrophins, signalling pathways, and functional peptide mimetics of neurotrophins and their receptors

SD Skaper - CNS & Neurological Disorders-Drug Targets …, 2008 - ingentaconnect.com
The neurotrophins are a family of closely related proteins that were first identified as survival
factors for sympathetic and sensory neurons, and have since been shown to control a …

A phase1 study of stereotactic gene delivery of AAV2-NGF for Alzheimer's disease

MS Rafii, TL Baumann, RAE Bakay, JM Ostrove… - Alzheimer's & …, 2014 - Elsevier
Background Nerve growth factor (NGF) is an endogenous neurotrophic-factor protein with
the potential to restore function and to protect degenerating cholinergic neurons in …

Postmortem analysis in a clinical trial of AAV2-NGF gene therapy for Alzheimer's disease identifies a need for improved vector delivery

MJ Castle, FC Baltanás, I Kovacs… - Human gene …, 2020 - liebertpub.com
Nerve growth factor (NGF) gene therapy rescues and stimulates cholinergic neurons, which
degenerate in Alzheimer's disease (AD). In a recent clinical trial for AD, intraparenchymal …

Gene therapy approach with an emphasis on growth factors: theoretical and clinical outcomes in neurodegenerative diseases

DGT Parambi, KS Alharbi, R Kumar, S Harilal… - Molecular …, 2022 - Springer
The etiology of many neurological diseases affecting the central nervous system (CNS) is
unknown and still needs more effective and specific therapeutic approaches. Gene therapy …

NGF is essential for hippocampal plasticity and learning

JM Conner, KM Franks, AK Titterness… - Journal of …, 2009 - Soc Neuroscience
Nerve growth factor (NGF) is produced in the hippocampus throughout life and is
retrogradely trafficked to septal cholinergic neurons, providing a potential mechanism for …