Review Article
Volume 14 Issue 7 - 2022
Odds of Gene Therapy: Current Approaches for the Treatment of Hitherto Not Curable Neurological Diseases
Gerhard Franz Walter*
International Neuroscience Institute Hannover, Germany
*Corresponding Author: Gerhard Franz Walter, International Neuroscience Institute Hannover, Germany.
Received: May 23, 2022; Published: June 28, 2022


In this review, principles of gene expression, patterns of inheritance and different types of mutations are recalled. The progress and expanding capacity of DNA-sequencing techniques for diagnostic genetic testing is the basis for the targeted treatment of genetic diseases by means of gene therapy. Cell transplantation and stem cell therapy, antisense oligonucleotide therapy, RNA interference for gene silencing, CRISPR/Cas9-mediated gene editing and read-through therapy are options for gene modification. Particle-mediated gene delivery, plasmid transfection and viral strategies using lentiviral vectors and adeno-associated virus vectors are options for gene delivery. These approaches alone or combined have the potential for the causal treatment of hitherto not curable neurological diseases.

Keywords: Cell Transplantation; Antisense Oligonucleotide Therapy; RNA Interference; CRISPR/Cas9-Mediated Gene Editing; Read- Through Therapy


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Citation: Gerhard Franz Walter. “Odds of Gene Therapy: Current Approaches for the Treatment of Hitherto Not Curable Neurological Diseases”. EC Neurology 14.7 (2022): 03-15.

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