Using Genome Editing for Alzheimer’s Disease Therapy: from Experiment to Clinic

Abstract

Here, we review the current achievements in the use of genome editing methods for experimental therapy of neurodegenerative diseases, primarily Alzheimer's disease. The main approaches used for genome editing and editing systems as well as their advantages and disadvantages are considered. Specific examples of the use of genome editing to correct mutations in genes associated with Alzheimer's disease are presented. The existing problems that need to be solved for the introduction of genome editing in the practice of treating neurodegenerative diseases are analyzed.

Emerging Strategies to Overcome Current CAR-T Therapy Dilemmas - Exosomes Derived from CAR-T Cells

“…Gene editing is a genetic manipulation technique that can modify DNA sequences at the genomic level, and it is currently used in experiments to treat neurological diseases (most notably Alzheimer's disease), which is feasible for future clinical use but has many issues that must be resolved before it can be used clinically. 115,116 This method still confronts numerous problems, including the non-negligible immunogenicity of fusion enzymes and delivery barriers, which must be overcome for clinical application to be effective. Fortunately, many reports of loading CRISPR/Cas9-like gene editing technologies into exosomes to address issues of transport and immunogenicity have been published, and increasing evidences suggest that exosomes could be used as an effective delivery vector for gene editing drugs and also for targeting cancers.…”