Retinitis Pigmentosa

Yu, Hyeong Gon

doi:10.1007/978-981-16-7337-5_4

Inherited Retinal Disease 2022

DOI: 10.1007/978-981-16-7337-5_4

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Retinitis Pigmentosa

Hyeong Gon Yu

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2024

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Cited by 1 publication

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Current developments of gene therapy in human diseases

Liu,

Li,

Zhu

et al. 2024

MedComm

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Gene therapy has witnessed substantial advancements in recent years, becoming a constructive tactic for treating various human diseases. This review presents a comprehensive overview of these developments, with a focus on their diverse applications in different disease contexts. It explores the evolution of gene delivery systems, encompassing viral (like adeno‐associated virus; AAV) and nonviral approaches, and evaluates their inherent strengths and limitations. Moreover, the review delves into the progress made in targeting specific tissues and cell types, spanning the eye, liver, muscles, and central nervous system, among others, using these gene technologies. This targeted approach is crucial in addressing a broad spectrum of genetic disorders, such as inherited lysosomal storage diseases, neurodegenerative disorders, and cardiovascular diseases. Recent clinical trials and successful outcomes in gene therapy, particularly those involving AAV and the clustered regularly interspaced short palindromic repeats (CRISPR)–CRISPR‐associated proteins, are highlighted, illuminating the transformative potentials of this approach in disease treatment. The review summarizes the current status of gene therapy, its prospects, and its capacity to significantly ameliorate patient outcomes and quality of life. By offering comprehensive analysis, this review provides invaluable insights for researchers, clinicians, and stakeholders, enriching the ongoing discourse on the trajectory of disease treatment.

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Current developments of gene therapy in human diseases

Liu,

Li,

Zhu

et al. 2024

MedComm

View full text Add to dashboard Cite

show abstract

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Retinitis Pigmentosa

Cited by 1 publication

References 209 publications

Current developments of gene therapy in human diseases

Current developments of gene therapy in human diseases

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