Cited by 55Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

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Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery

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Joshi, Chaitanya R., Labhasetwar, Vinod, Ghorpade, Anuja·2017·Journal of Neuroimmune Pharmacology

DOI 10.1007/s11481-016-9724-3PMC PMC5393046

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Abstract

Neurological diseases and disorders (NDDs) present a significant societal burden and currently available drug- and biological-based therapeutic strategies have proven inadequate to alleviate it. Gene therapy is a suitable alternative to treat NDDs compared to conventional systems since it can be tailored to specifically alter select gene expression, reverse disease phenotype and restore normal function. The scope of gene therapy has broadened over the years with the advent of RNA interference and genome editing technologies. Consequently, encouraging results from central nervous system (CNS)-targeted gene delivery studies have led to their transition from preclinical to clinical trials. As we shift to an exciting gene therapy era, a retrospective of available literature on CNS-associated gene delivery is in order. This review is timely in this regard, since it analyzes key challenges and major findings from the last two decades and evaluates future prospects of brain gene delivery. We emphasize major areas consisting of physiological and pharmacological challenges in gene therapy, function-based selection of a ideal cellular target(s), available therapy modalities, and diversity of viral vectors and nanoparticles as vehicle systems. Further, we present plausible answers to key questions such as strategies to circumvent low blood-brain barrier permeability and most suitable CNS cell types for targeting. We compare and contrast pros and cons of the tested viral vectors in the context of delivery systems used in past and current clinical trials. Gene vector design challenges are also evaluated in the context of cell-specific promoters. Key challenges and findings reported for recent gene therapy clinical trials, assessing viral vectors and nanoparticles are discussed from the perspective of bench to bedside gene therapy translation. We conclude this review by tying together gene delivery challenges, available vehicle systems and comprehensive analyses of neuropathogenesis to outline future prospects of CNS-targeted gene therapies.

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See all 55

Authors: Joshi, C. R., Labhasetwar, V., & Ghorpade, A.
Affiliations: University of North Texas Health Science Center (US)
Cleveland Clinic (US)
Corresponding author: Ghorpade, Anuja
ORCID: Joshi, Chaitanya R.
Labhasetwar, Vinod
Venue: Journal of Neuroimmune Pharmacology
Publisher: Springer Science and Business Media LLC
Published: 2017-02-03
Volume: 12
Issue: 1
Pages: 51-83
ISSN: 1557-1890 (ISSN-L)
1557-1904
DOI: 10.1007/s11481-016-9724-3
PMC: PMC5393046
OpenAlex: W2585557706
Cited by: 55
References: 259
Citations by year: 2025: 4 · 2024: 8 · 2023: 4 · 2022: 2 · 2021: 5 · 2020: 10 · 2019: 5 · 2018: 10 · 2017: 7
FWCI: 1.13
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springernature.com/gp/researchers/text-and-data-mining (tdm)
www.springernature.com/gp/researchers/text-and-data-mining (vor)
Locations: Journal of Neuroimmune Pharmacology (publishedVersion)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Institute of Neurological Disorders and Stroke (NS048837, NS070896)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
Keywords: gene therapy, gene delivery to the brain, central nervous system, neurological disorders, blood-brain barrier, viral vectors, nanoparticles, RNA interference, genome editing, cell-specific promoters, clinical trials, translational medicine, neuropathogenesis
MeSH terms: Animals, Blood-Brain Barrier, Brain, Forecasting, Humans, Genetic Therapy, Gene Transfer Techniques, Neurodegenerative Diseases

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Destination Brain: the Past, Present, and Future of Therapeutic Gene Delivery

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