Genome editing in the treatment of ocular diseases

Choi, Elliot H.; Suh, Susie; Sears, Avery E.; Hołubowicz, Rafał; Kedhar, Sanjay R.; Browne, Andrew W.; Palczewski, Krzysztof

doi:10.1038/s12276-023-01057-2

Cited by 6 publications

(1 citation statement)

References 199 publications

(228 reference statements)

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“…Researchers are exploring ways to deliver therapeutic genes to affect such neurodegenerative diseases [ 52 ]. Furthermore, some gene therapies, including genome editing technologies, have been suggested for both inherited and non-inherited retinal diseases in humans [ 53 ]. Gene therapy is also promising in veterinary medicine, for example, in therapy for cancer, muscular dystrophy, or various hereditary pathophysiologies [ 54 , 55 ].…”

Section: Applications Of Gene Therapy In Human and Veterinary Medicinementioning

confidence: 99%

Epigenetic and gene therapy in human and veterinary medicine

Bártová

2024

Environmental Epigenetics

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Gene therapy is a focus of interest in both human and veterinary medicine, especially in recent years due to the potential applications of CRISPR/Cas9 technology. Another relatively new approach is that of epigenetic therapy, which involves an intervention based on epigenetic marks, including DNA methylation, histone post-translational modifications, and post-transcription modifications of distinct RNAs. The epigenome results from enzymatic reactions which regulate gene expression without altering DNA sequences. In contrast to conventional CRISP/Cas9 techniques, the recently established methodology of epigenetic editing mediated by the CRISPR/dCas9 system is designed to target specific genes without causing DNA breaks. Both natural epigenetic processes and epigenetic editing regulate gene expression and thereby contribute to maintaining the balance between physiological functions and pathophysiological states. From this perspective, knowledge of specific epigenetic marks has immense potential in both human and veterinary medicine. For instance, the use of epigenetic drugs (chemical compounds with therapeutic potential affecting the epigenome) seems to be promising for the treatment of cancer, metabolic, and infectious diseases. Also, there is evidence that an epigenetic diet (nutrition-like factors affecting epigenome) should be considered as part of a healthy lifestyle and could contribute to the prevention of pathophysiological processes. In summary, epigenetic-based approaches in human and veterinary medicine have increasing significance in targeting aberrant gene expression associated with various diseases. In this case, CRISPR/dCas9, epigenetic targeting, and some epigenetic nutrition factors could contribute to reversing an abnormal epigenetic landscape to a healthy physiological state.

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Section: Applications Of Gene Therapy In Human and Veterinary Medicinementioning

confidence: 99%

Epigenetic and gene therapy in human and veterinary medicine

Bártová

2024

Environmental Epigenetics

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Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment

Yang,

Bui,

Mei

et al. 2024

Adv Funct Materials

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Human genetic disorders, arising from a range of genetic irregularities, can significantly affect human physiology, often with limited available treatment options. The development of the CRISPR system, facilitating precise editing of the genome, has opened new avenues for addressing a range of mutations found in various genetic disorders. However, there is currently a lack of comprehensive reviews that specifically address the application of CRISPR in genetic diseases. To bridge this gap, this review focuses on exploring the advancements in CRISPR technology and their utility in therapeutic approaches for various genetic disorders. This review introduces human genetic disorders, explains the fundamental mechanisms of CRISPR editing, and highlights the latest advancements in CRISPR technology. Additionally, it examines three CRISPR delivery techniques, including physical delivery, viral vectors, and nanocarriers. It further reviews CRISPR's applications in therapeutic approaches for genetic disorders. Finally, it identifies the primary hurdles associated with industrial development and ethics considerations that should be addressed before the application of CRISPR in a medical context.

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