Abstract
To date, gene therapy has employed viral vectors to deliver therapeutic genes. However, recent progress in molecular and cell biology has revolutionized the field of stem cells and gene therapy. A few years ago, clinical trials started using stem cell replacement therapy, and the induced pluripotent stem cells (iPSCs) technology combined with CRISPR-Cas9 gene editing has launched a new era in gene therapy for the treatment of neurological disorders. Here, we summarize the latest findings in this research field and discuss their clinical applications, emphasizing the relevance of recent studies in the development of innovative stem cell and gene editing therapeutic approaches. Even though tumorigenicity and immunogenicity are existing hurdles, we report how recent progress has tackled them, making engineered stem cell transplantation therapy a realistic option.
Highlights
Treatment of Neurological Diseases.Nearly ten years have passed since the Nobel prize was awarded to Shinya Yamanaka for having successfully reprogrammed somatic cells into pluripotent stem cells [1,2]
IPSCs represent an indefinite source of patient-derived cells [5]. induced pluripotent stem cells (iPSCs) technology resulted useful for understanding the pathophysiology of human neurological disorders difficult to study because of the inaccessibility of the tissue of interest, as the nervous system
On the contrary, gene therapy approaches targeting the central nervous system are very recent and for this reason we will focus on iPSCs and gene therapy in neurological disorders
Summary
A CRISPR Guide Assisted Reduction of Damage (CRISPR GUARD) co-delivering short RNAs to guide the CRISPR-Cas system, minimizing off-target mutagenesis, has been recently developed [10] These latest advances together with the iPSC technology have set the stage for a permanent and efficient treatment for neurological disorders. Represents the Generation Gene Therapy, hopefully to be considered as the gold standard therapy in the near future Ever since their first findings, the work performed by Yamanaka and coworkers has focused many research efforts on stem cells, on retinal degeneration diseases, and on other neurological disorders [13].
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