Recent gene therapy advancements for neurological diseases

Sahana Nagabhushan Kalburgi, Nadia N. Khan, Steven J. Gray

Research output: Contribution to journalArticle

23 Citations (Scopus)

Abstract

The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous system and modest successes in human gene therapy trials. The purpose of this review is to describe commonly-used viral gene transfer vectors and recent advancements towards producing meaningful gene-based treatments for central nervous system (CNS) disorders. Gene therapy trials for Canavan disease, Batten disease, adrenoleukodystrophy, and Parkinson's disease are discussed to illustrate the current state of clinical gene transfer to the CNS. Preclinical studies are under way for a number of diseases, primarily lysosomal storage disorders, using a newer generation of vectors and delivery strategies. Relevant studies in animal models are highlighted for Mucopolysaccharidosis IIIB and Krabbe disease to provide a prelude for what can be expected in the coming years for human gene transfer trials, using recent advancements in gene transfer technology. In conclusion, recent improvements in CNS gene transfer technology are expected to significantly increase the degree of disease rescue in future CNS-directed clinical trials, exceeding the modest clinical successes that have been observed so far.

Original languageEnglish (US)
Pages (from-to)111-119
Number of pages9
JournalDiscovery medicine
Volume15
Issue number81
StatePublished - Oct 29 2013

Fingerprint

Genetic Therapy
Genes
Technology Transfer
Central Nervous System
Canavan Disease
Globoid Cell Leukodystrophy
Adrenoleukodystrophy
Neuronal Ceroid-Lipofuscinoses
Mucopolysaccharidoses
Lysosomal Storage Diseases
Viral Genes
Central Nervous System Diseases
Nervous System
Parkinson Disease
Animal Models
Clinical Trials

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Recent gene therapy advancements for neurological diseases. / Kalburgi, Sahana Nagabhushan; Khan, Nadia N.; Gray, Steven J.

In: Discovery medicine, Vol. 15, No. 81, 29.10.2013, p. 111-119.

Research output: Contribution to journalArticle

Kalburgi, SN, Khan, NN & Gray, SJ 2013, 'Recent gene therapy advancements for neurological diseases', Discovery medicine, vol. 15, no. 81, pp. 111-119.
Kalburgi, Sahana Nagabhushan ; Khan, Nadia N. ; Gray, Steven J. / Recent gene therapy advancements for neurological diseases. In: Discovery medicine. 2013 ; Vol. 15, No. 81. pp. 111-119.
@article{283d2ee7825347eaa8c2db0481a3aa2a,
title = "Recent gene therapy advancements for neurological diseases",
abstract = "The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous system and modest successes in human gene therapy trials. The purpose of this review is to describe commonly-used viral gene transfer vectors and recent advancements towards producing meaningful gene-based treatments for central nervous system (CNS) disorders. Gene therapy trials for Canavan disease, Batten disease, adrenoleukodystrophy, and Parkinson's disease are discussed to illustrate the current state of clinical gene transfer to the CNS. Preclinical studies are under way for a number of diseases, primarily lysosomal storage disorders, using a newer generation of vectors and delivery strategies. Relevant studies in animal models are highlighted for Mucopolysaccharidosis IIIB and Krabbe disease to provide a prelude for what can be expected in the coming years for human gene transfer trials, using recent advancements in gene transfer technology. In conclusion, recent improvements in CNS gene transfer technology are expected to significantly increase the degree of disease rescue in future CNS-directed clinical trials, exceeding the modest clinical successes that have been observed so far.",
author = "Kalburgi, {Sahana Nagabhushan} and Khan, {Nadia N.} and Gray, {Steven J.}",
year = "2013",
month = "10",
day = "29",
language = "English (US)",
volume = "15",
pages = "111--119",
journal = "Discovery medicine",
issn = "1539-6509",
publisher = "Discovery Medicine",
number = "81",

}

TY - JOUR

T1 - Recent gene therapy advancements for neurological diseases

AU - Kalburgi, Sahana Nagabhushan

AU - Khan, Nadia N.

AU - Gray, Steven J.

PY - 2013/10/29

Y1 - 2013/10/29

N2 - The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous system and modest successes in human gene therapy trials. The purpose of this review is to describe commonly-used viral gene transfer vectors and recent advancements towards producing meaningful gene-based treatments for central nervous system (CNS) disorders. Gene therapy trials for Canavan disease, Batten disease, adrenoleukodystrophy, and Parkinson's disease are discussed to illustrate the current state of clinical gene transfer to the CNS. Preclinical studies are under way for a number of diseases, primarily lysosomal storage disorders, using a newer generation of vectors and delivery strategies. Relevant studies in animal models are highlighted for Mucopolysaccharidosis IIIB and Krabbe disease to provide a prelude for what can be expected in the coming years for human gene transfer trials, using recent advancements in gene transfer technology. In conclusion, recent improvements in CNS gene transfer technology are expected to significantly increase the degree of disease rescue in future CNS-directed clinical trials, exceeding the modest clinical successes that have been observed so far.

AB - The past few years have seen rapid advancements in vector-mediated gene transfer to the nervous system and modest successes in human gene therapy trials. The purpose of this review is to describe commonly-used viral gene transfer vectors and recent advancements towards producing meaningful gene-based treatments for central nervous system (CNS) disorders. Gene therapy trials for Canavan disease, Batten disease, adrenoleukodystrophy, and Parkinson's disease are discussed to illustrate the current state of clinical gene transfer to the CNS. Preclinical studies are under way for a number of diseases, primarily lysosomal storage disorders, using a newer generation of vectors and delivery strategies. Relevant studies in animal models are highlighted for Mucopolysaccharidosis IIIB and Krabbe disease to provide a prelude for what can be expected in the coming years for human gene transfer trials, using recent advancements in gene transfer technology. In conclusion, recent improvements in CNS gene transfer technology are expected to significantly increase the degree of disease rescue in future CNS-directed clinical trials, exceeding the modest clinical successes that have been observed so far.

UR - http://www.scopus.com/inward/record.url?scp=84886290228&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84886290228&partnerID=8YFLogxK

M3 - Article

C2 - 23449113

AN - SCOPUS:84886290228

VL - 15

SP - 111

EP - 119

JO - Discovery medicine

JF - Discovery medicine

SN - 1539-6509

IS - 81

ER -