Gene therapy tools for brain diseases

S Ingusci, G Verlengia, M Soukupova… - Frontiers in …, 2019 - frontiersin.org
Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …

Prospects for new restorative and neuroprotective treatments in Parkinson's disease

SB Dunnett, A Björklund - Nature, 1999 - nature.com
The degeneration of forebrain dopamine systems in Parkinson's disease has been an
effective target for pharmaceutical research over the past four decades. However, although …

CNS plasticity and assessment of forelimb sensorimotor outcome in unilateral rat models of stroke, cortical ablation, parkinsonism and spinal cord injury

T Schallert, SM Fleming, JL Leasure, JL Tillerson… - …, 2000 - Elsevier
We have reviewed a battery of useful tests for evaluating sensorimotor function and plasticity
acutely and chronically in unilateral rat models of central nervous system injury. These tests …

Pharmacological validation of behavioural measures of akinesia and dyskinesia in a rat model of Parkinson's disease

M Lundblad, M Andersson, C Winkler… - European Journal of …, 2002 - Wiley Online Library
In an attempt to define clinically relevant models of akinesia and dyskinesia in 6‐
hydroxydopamine (6‐OHDA)‐lesioned rats, we have examined the effects of drugs with high …

Transplants of fibroblasts genetically modified to express BDNF promote regeneration of adult rat rubrospinal axons and recovery of forelimb function

Y Liu, D Kim, BT Himes, SY Chow… - Journal of …, 1999 - Soc Neuroscience
Adult mammalian CNS neurons do not normally regenerate their severed axons. This failure
has been attributed to scar tissue and inhibitory molecules at the injury site that block the …

Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the …

D Kirik, C Rosenblad, A Björklund… - Journal of …, 2000 - Soc Neuroscience
Previous studies have used recombinant adeno-associated viral (rAAV) vectors to deliver
glial cell line-derived neurotrophic factor (GDNF) in the substantia nigra to protect the nigral …

Forced limb-use effects on the behavioral and neurochemical effects of 6-hydroxydopamine

JL Tillerson, AD Cohen, J Philhower… - Journal of …, 2001 - Soc Neuroscience
Rats with unilateral depletion of striatal dopamine (DA) show marked preferential use of the
ipsilateral forelimb. Previous studies have shown that implementation of motor therapy after …

Regulatable gene expression systems for gene therapy applications: progress and future challenges

S Goverdhana, M Puntel, W Xiong, JM Zirger, C Barcia… - Molecular Therapy, 2005 - cell.com
Gene therapy aims to revert diseased phenotypes by the use of both viral and nonviral gene
delivery systems. Substantial progress has been made in making gene transfer vehicles …

Clinicopathological findings following intraventricular glial‐derived neurotrophic factor treatment in a patient with Parkinson's disease

JH Kordower, S Palfi, EY Chen, SY Ma… - Annals of Neurology …, 1999 - Wiley Online Library
As part of a safety and tolerability study, a 65‐year‐old man with Parkinson's disease (PD)
received monthly intracerebroventricular injections of glial‐derived neurotrophic factor …

Towards a neuroprotective gene therapy for Parkinson's disease: use of adenovirus, AAV and lentivirus vectors for gene transfer of GDNF to the nigrostriatal system in …

A Björklund, D Kirik, C Rosenblad, B Georgievska… - Brain research, 2000 - Elsevier
During the last few years, recombinant viral vectors derived from adenovirus (Ad), adeno-
associated virus (AAV) or lentivirus (LV) have been developed into highly effective vehicles …