CRISPR/Cas9 gene editing: A new hope for transthyretin amyloidosis treatment

Shahzad, Faizan; Rehman, Mohammad Ebad Ur; Basit, Jawad; Saeed, Sajeel; Abbas, Khawar; Farhan, Muhammad

doi:10.1016/j.amsu.2022.104784

Cited by 4 publications

(2 citation statements)

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“…This mutation has been shown to lower β-secretase cleavage by 40% [97]. As a result of the previous research, inserting the A673T mutation into patient's neurons might be an effective and long-term the strategy of reducing the risk factor for patients with Alzheimer's disease [91]. To that goal, they used a CRISPR/Cas9-based method to change the APP gene in HEK293T and SH-SY5Y cells by transforming the alanine codon to a threonine (containing the APP gene with deaminated cytosine1 and cytosine2 positions).…”

Section: Crispr/cas9 Potential In Ad: a Promising Role Of Crispr/cas9...mentioning

confidence: 94%

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A Review of CRISPR Cas9 for Alzheimer’s Disease: Treatment Strategies and Could target APOE e4, APP, and PSEN-1 Gene using CRISPR cas9 Prevent the Patient from Alzheimer’s Disease?

Adji

Widjaja²,

Wardani³

et al. 2022

Open Access Maced J Med Sci

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A Review of CRISPR Cas9 for Alzheimer’s Disease: Treatment Strategies and Could target APOE e4, APP, and PSEN-1 Gene using CRISPR cas9 Prevent the Patient from Alzheimer’s Disease? BACKGROUND: Alzheimer’s disease is a neurodegenerative disorder characterized by the formation of β-amyloid plaques and neurofibrillary tangles from hyperphosphorylated tau. Several studies suggest that targeting the deletion of the APOE e4, PSEN-1, and APP will reduce tau phosphorylation and Aβ protein accumulation, a crucial hypothesis for the causation of Alzheimer’s disease. APOE e4, PSEN-1, and APP with genome editing Clustered Regular interspersed Short Palindromic Repeats-CRISPR-related (CRISPR/Cas9) are thought to have therapeutic promise for Alzheimer’s disease.AIM: The purpose of this study was to determine whether targeting APOE e4, PSEN-1, and APP using CRISPR/Cas9 is an effective therapeutic and whether it has a long-term effect on Alzheimer’s disease.METHODS: The method used in this study summarized articles by examining the titles and abstracts of specific specified keywords. In this situation, the author picked the title and abstract that matched PubMed, Google Scholar, Science Direct, Cochrane, and the Frontiers in Neuroscience; this was followed by checking to see whether the paper was available in full-text. Eventually, the researcher will study the entire article to decide if it is valuable and relevant to the issue.RESULTS: CRISPR/Cas9 deletion of APOE e4, PSEN-1, and APP in induced pluripotent stem cells (iPSC’s) and g2576 mice as APP mutant models reduce tau phosphorylation and Aβ protein accumulation from neurofibrillary tangles and prevent cell death, vascular damage, and dementia. Furthermore, CRISPR/Cas9 deletion in APOE e4, PSEN-1, and APP improved neuronal cell resilience to oxidative stress and inflammation.CONCLUSION: APOE e4, PSEN-1, and APP deletion by genome editing CRISPR/Cas9 is effective to reduce tau phosphorylation and Aβ protein accumulation from neurofibrillary tangles, cell death, vascular damage, and dementia. However, further research is needed to determine the side effects and safety of its use.

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Section: Crispr/cas9 Potential In Ad: a Promising Role Of Crispr/cas9...mentioning

confidence: 94%

“…In addition, the CRISPR/Cas9 system can also be used as programmable antibiotics to kill the bacteria sequence specifically and, therefore, can bypass multidrug resistance. Furthermore, CRISPR/Cas9-based gene drive may also hold the potential to limit the spread of vector-borne diseases [58], [91].…”

Section: Crispr/cas 9: a Promising Gene-editing Toolmentioning

confidence: 99%