Parkinson's disease gene therapy: success by design meets failure by efficacy

RT Bartus, MS Weinberg, RJ Samulski - Molecular Therapy, 2014 - cell.com
Over the past decade, nine gene therapy clinical trials for Parkinson's disease (PD) have
been initiated and completed. Starting with considerable optimism at the initiation of each …

New molecular therapies for the treatment of hearing loss

Y Ma, AK Wise, RK Shepherd… - Pharmacology & …, 2019 - Elsevier
An estimated 466 million people suffer from hearing loss worldwide. Sensorineural hearing
loss is characterized by degeneration of key structures of the sensory pathway in the …

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 …

Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients

RT Bartus, TL Baumann, J Siffert, CD Herzog… - Neurology, 2013 - AAN Enterprises
Objective: In an effort to account for deficiencies in axonal transport that limit the
effectiveness of neurotrophic factors, this study tested the safety and feasibility, in …

Calming the Nerves via the Immune Instructive Physiochemical Properties of Self‐Assembling Peptide Hydrogels

N Mahmoudi, E Mohamed, SS Dehnavi… - Advanced …, 2024 - Wiley Online Library
Current therapies for the devastating damage caused by traumatic brain injuries (TBI) are
limited. This is in part due to poor drug efficacy to modulate neuroinflammation …

Post-mortem assessment of the short and long-term effects of the trophic factor neurturin in patients with α-synucleinopathies

RT Bartus, JH Kordower, EM Johnson Jr, L Brown… - Neurobiology of …, 2015 - Elsevier
Substantial interest persists for developing neurotrophic factors to treat neurodegenerative
diseases. At the same time, significant progress has been made in implementing gene …

Progress and prospects of gene therapy clinical trials for the muscular dystrophies

NE Bengtsson, JT Seto, JK Hall… - Human molecular …, 2016 - academic.oup.com
Clinical trials represent a critical avenue for new treatment development, where early
phases (I, I/II) are designed to test safety and effectiveness of new therapeutics or diagnostic …

Advancing neurotrophic factors as treatments for age-related neurodegenerative diseases: developing and demonstrating “clinical proof-of-concept” for AAV-neurturin …

RT Bartus, TL Baumann, L Brown, BR Kruegel… - Neurobiology of …, 2013 - Elsevier
Neurotrophic factors have long shown promise as potential therapies for age-related
neurodegenerative diseases. However, 20 years of largely disappointing clinical results …

Delivery of neurotrophic factors in the treatment of age-related chronic neurodegenerative diseases

S Padmakumar, MS Taha, E Kadakia… - Expert opinion on …, 2020 - Taylor & Francis
Introduction Neurodegenerative diseases are those wherein the neurons in the brain or
peripheral nervous system lose their function, eventually culminating in neuronal death …

Divergent total syntheses of lyconadins A and C

Y Yang, CW Haskins, W Zhang… - Angewandte Chemie …, 2014 - Wiley Online Library
Divergent and concise total syntheses of two lycopodium alkaloids, lyconadins A and C
have been developed. The synthesis of lyconadin A, having potent neurotrophic activity …