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 …
been initiated and completed. Starting with considerable optimism at the initiation of each …
New molecular therapies for the treatment of hearing loss
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 …
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 …
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 …
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
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 …
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 …
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 …
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 …
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 …
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 …
have been developed. The synthesis of lyconadin A, having potent neurotrophic activity …