CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics

Bhardwaj, Shanu; Kesari, Kavindra Kumar; Rachamalla, Mahesh; Mani, Shalini; Ashraf, Ghulam Md; Jha, Saurabh; Kumar, Pravir; Ambasta, Rashmi K.; Dureja, Harish; Devkota, Hari Prasad; Gupta, Gaurav; Chellappan, Dinesh Kumar; Singh, Sachin Kumar; Dua, Kamal; Ruokolainen, Janne; Kamal, Mohammad Amjad; Ojha, Shreesh; Jha, Niraj Kumar

doi:10.1016/j.jare.2021.07.001

Cited by 67 publications

(24 citation statements)

References 95 publications

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“…In addition, CRISPR/Cas9 technology is also widely used for bone regeneration and the treatment of CFTR, Alzheimer's disease, obesity, and other diseases 72,73,192,[281][282][283][284][285] (Fig. 6d).…”

Section: Attempts To Treat Diseasesmentioning

confidence: 99%

CRISPR/Cas9 therapeutics: progress and prospects

Yang

et al. 2023

Sig Transduct Target Ther

223

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Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases by permanently correcting deleterious base mutations or disrupting disease-causing genes with great precision and efficiency. A variety of efficient Cas9 variants and derivatives have been developed to cope with the complex genomic changes that occur during diseases. However, strategies to effectively deliver the CRISPR system to diseased cells in vivo are currently lacking, and nonviral vectors with target recognition functions may be the focus of future research. Pathological and physiological changes resulting from disease onset are expected to serve as identifying factors for targeted delivery or targets for gene editing. Diseases are both varied and complex, and the choice of appropriate gene-editing methods and delivery vectors for different diseases is important. Meanwhile, there are still many potential challenges identified when targeting delivery of CRISPR/Cas9 technology for disease treatment. This paper reviews the current developments in three aspects, namely, gene-editing type, delivery vector, and disease characteristics. Additionally, this paper summarizes successful examples of clinical trials and finally describes possible problems associated with current CRISPR applications.

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Section: Attempts To Treat Diseasesmentioning

confidence: 99%

CRISPR/Cas9 therapeutics: progress and prospects

Yang

et al. 2023

Sig Transduct Target Ther

223

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“…These diseases are mainly characterized by loss in the functioning of neurons and the formation of misfolded proteins, accompanied by their aggregation, extracellular intracellular deposits, and neuronal cell death. Excessive release of reactive oxygen species (ROS) and neuroinflammation contribute chiefly to the pathophysiology of different neurodegenerative diseases and depict a direct consequence of perturbation in the homeostasis of the central nervous system (CNS) [ 67 ]. Effective treatments are being explored, and efforts are being made to manage age-related degeneration and promote stability in patients.…”

Section: Marine Drugs In Neurodegenerationmentioning

confidence: 99%

Biotechnological Innovations from Ocean: Transpiring Role of Marine Drugs in Management of Chronic Disorders

et al. 2022

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Marine drugs are abundant in number, comprise of a diverse range of structures with corresponding mechanisms of action, and hold promise for the discovery of new and better treatment approaches for the management of several chronic diseases. There are huge reserves of natural marine biological compounds, as 70 percent of the Earth is covered with oceans, indicating a diversity of chemical entities on the planet. The marine ecosystems are a rich source of bioactive products and have been explored for lead drug molecules that have proven to be novel therapeutic targets. Over the last 70 years, many structurally diverse drug products and their secondary metabolites have been isolated from marine sources. The drugs obtained from marine sources have displayed an exceptional potential in the management of a wide array of diseases, ranging from acute to chronic conditions. A beneficial role of marine drugs in human health has been recently proposed. The current review highlights various marine drugs and their compounds and role in the management of chronic diseases such as cancer, diabetes, neurodegenerative diseases, and cardiovascular disorders, which has led to the development of new drug treatment approaches.

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“…CRISPR/Cas9 based gene-editing: This approach has demonstrated a great potential for the treatment of AD and other diseases by correcting specific gene sequences ( DiCarlo et al, 2017 ; Schneller et al, 2017 ; German et al, 2019 ; Mirza and Karim, 2019 ; Karimian et al, 2020 ; Xu et al, 2020 ; Bhardwaj et al, 2021 ). In a study using CRISPR/Cas9, gene editing of endogenous APP at the extreme C-terminus inhibited interactions of APP and BAC1 within the endosomes, thereby the most important cleavage event in Aβ generation was avoided in Sporadic AD ( Das et al, 2013 ; Sun et al, 2019 ).…”

Section: Therapeutic Prospects For the Treatment Of Alzheimer’s Diseasementioning

confidence: 99%

Clinical relevance of biomarkers, new therapeutic approaches, and role of post-translational modifications in the pathogenesis of Alzheimer’s disease

Mumtaz

Ayaz

Khan

et al. 2022

Front. Aging Neurosci.

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Alzheimer’s disease (AD) is a neurodegenerative disorder that causes progressive loss of cognitive functions like thinking, memory, reasoning, behavioral abilities, and social skills thus affecting the ability of a person to perform normal daily functions independently. There is no definitive cure for this disease, and treatment options available for the management of the disease are not very effective as well. Based on histopathology, AD is characterized by the accumulation of insoluble deposits of amyloid beta (Aβ) plaques and neurofibrillary tangles (NFTs). Although several molecular events contribute to the formation of these insoluble deposits, the aberrant post-translational modifications (PTMs) of AD-related proteins (like APP, Aβ, tau, and BACE1) are also known to be involved in the onset and progression of this disease. However, early diagnosis of the disease as well as the development of effective therapeutic approaches is impeded by lack of proper clinical biomarkers. In this review, we summarized the current status and clinical relevance of biomarkers from cerebrospinal fluid (CSF), blood and extracellular vesicles involved in onset and progression of AD. Moreover, we highlight the effects of several PTMs on the AD-related proteins, and provide an insight how these modifications impact the structure and function of proteins leading to AD pathology. Finally, for disease-modifying therapeutics, novel approaches, and targets are discussed for the successful treatment and management of AD.

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CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics

Cited by 67 publications

References 95 publications

CRISPR/Cas9 therapeutics: progress and prospects

CRISPR/Cas9 therapeutics: progress and prospects

Biotechnological Innovations from Ocean: Transpiring Role of Marine Drugs in Management of Chronic Disorders

Clinical relevance of biomarkers, new therapeutic approaches, and role of post-translational modifications in the pathogenesis of Alzheimer’s disease

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