CRISPR-Cas9 in Alzheimer’s disease: Therapeutic trends, modalities, and challenges

CRISPR-Cas9 in Alzheimer’s disease

The application of CRISPR-Cas9 genome editing for the treatment of AD has run into several challenges. The lack of an efficient delivery mechanism could further lead to an unfavorable immune response in AD patients as well as fatal outcomes. On the other hand, future advances that can potentially increase the effectiveness of CRISPR-Cas9 in AD therapies are anticipated on a number of fronts. In addition to research on finding an accurate in vitro model, one anticipated focus is the development of an appropriate AD animal model that could effectively and equivalently replicate the onset and progression of AD pathology like the human brain. Recent strategies have also come up with the prospect of overturning the risks of off-target trimming at unspecified genomic locations, genetic mosaicism, and potential epigenetic effects. Further research on improving delivery tools and genome editing time, negotiating toxicity issues, and managing the therapeutic cost could promote clinical and translational applications of CRISPR-Cas9 gene editing to AD therapeutics. Finally, initiatives such as identifying genetic/molecular markers and potential genomic targets for AD, assessing viral vectors to introduce mutations, and employing safe non-viral vectors for gene-editing maneuvers could inspire progress toward finding an effective breakthrough in AD therapeutics by employing CRISPR Cas9 genome-editing machinery https://ars.els-cdn.com/content/image/1-s2.0-S135964462300168X-ga1_lrg.jpg