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Next Generation Precision Medicine: CRISPR-mediated Genome Editing for the Treatment of Neurodegenerative Disorders
- Source :
- J Neuroimmune Pharmacol
- Publication Year :
- 2019
- Publisher :
- Springer Science and Business Media LLC, 2019.
-
Abstract
- Despite significant advancements in the field of molecular neurobiology especially neuroinflammation and neurodegeneration, the highly complex molecular mechanisms underlying neurodegenerative diseases remain elusive. As a result, the development of the next generation neurotherapeutics has experienced a considerable lag phase. Recent advancements in the field of genome editing offer a new template for dissecting the precise molecular pathways underlying the complex neurodegenerative disorders. We believe that the innovative genome and transcriptome editing strategies offer an excellent opportunity to decipher novel therapeutic targets, develop novel neurodegenerative disease models, develop neuroimaging modalities, develop next-generation diagnostics as well as develop patient-specific precision-targeted personalized therapies to effectively treat neurodegenerative disorders including Alzheimer's disease, Parkinson's disease, Huntington's disease, Amyotrophic lateral sclerosis, Frontotemporal dementia etc. Here, we review the latest developments in the field of CRISPR-mediated genome editing and provide unbiased futuristic insights regarding its translational potential to improve the treatment outcomes and minimize financial burden. However, despite significant advancements, we would caution the scientific community that since the CRISPR field is still evolving, currently we do not know the full spectrum of CRISPR-mediated side effects. In the wake of the recent news regarding CRISPR-edited human babies being born in China, we urge the scientific community to maintain high scientific and ethical standards and utilize CRISPR for developing in vitro disease in a dish model, in vivo testing in nonhuman primates and lower vertebrates and for the development of neurotherapeutics for the currently incurable neurodegenerative disorders. Graphical Abstract.
- Subjects :
- 0301 basic medicine
Immunology
Neuroscience (miscellaneous)
Disease
Article
03 medical and health sciences
0302 clinical medicine
Genome editing
Huntington's disease
CRISPR-Associated Protein 9
Animals
Humans
Immunology and Allergy
Medicine
CRISPR
Precision Medicine
Gene Editing
Pharmacology
business.industry
Neurodegeneration
Neurodegenerative Diseases
Genetic Therapy
medicine.disease
Precision medicine
Treatment Outcome
030104 developmental biology
DECIPHER
CRISPR-Cas Systems
business
Neuroscience
030217 neurology & neurosurgery
Frontotemporal dementia
Subjects
Details
- ISSN :
- 15571904 and 15571890
- Volume :
- 14
- Database :
- OpenAIRE
- Journal :
- Journal of Neuroimmune Pharmacology
- Accession number :
- edsair.doi.dedup.....3172c1032796e04519b362cbac67ebbc