Cargando…

Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives

Advancements in genome editing enable permanent changes of DNA sequences in a site-specific manner, providing promising approaches for treating human genetic disorders caused by gene mutations. Recently, genome editing has been applied and achieved significant progress in treating inherited genetic...

Descripción completa

Detalles Bibliográficos
Autores principales:	Huang, Chao, Li, Qing, Li, Jinsong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	De Gruyter 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10388762/ https://www.ncbi.nlm.nih.gov/pubmed/37724161 http://dx.doi.org/10.1515/mr-2022-0029

Ejemplares similares

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
por: Hansen, Silja, et al.
Publicado: (2023)

Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies
por: Nguyen, Quynh, et al.
Publicado: (2020)

Prime Editing for Inherited Retinal Diseases
por: da Costa, Bruna Lopes, et al.
Publicado: (2021)

In utero nanoparticle delivery for site-specific genome editing
por: Ricciardi, Adele S., et al.
Publicado: (2018)

CRISPR/Cas9 genome editing technology in filamentous fungi: progress and perspective
por: Song, Runjie, et al.
Publicado: (2019)

Gene Editing for Inherited Red Blood Cell Diseases
por: Quintana-Bustamante, Oscar, et al.
Publicado: (2022)

Toward the Treatment of Inherited Diseases of the Retina Using CRISPR-Based Gene Editing
por: Hernández-Juárez, Jennifer, et al.
Publicado: (2021)

Improving the efficiency of precise genome editing with site-specific Cas9-oligonucleotide conjugates
por: Ling, Xinyu, et al.
Publicado: (2020)

Genome Sequence-Independent Identification of RNA Editing Sites
por: Zhang, Qing, et al.
Publicado: (2015)

Inherited cardiac diseases, pluripotent stem cells, and genome editing combined—the past, present, and future
por: van den Brink, Lettine, et al.
Publicado: (2019)

The Potential of CRISPR/Cas9 Gene Editing as a Treatment Strategy for Inherited Diseases
por: Abdelnour, Sameh A., et al.
Publicado: (2021)

Current Progress of Mitochondrial Genome Editing by CRISPR
por: Yin, Tao, et al.
Publicado: (2022)

Preface to the special topic on genome editing research in China
por: Li, Jinsong, et al.
Publicado: (2019)

Efficient CRISPR‐based genome editing using tandem guide RNAs and editable surrogate reporters
por: Liu, Wuqing, et al.
Publicado: (2018)

An Editing-Site-Specific PCR Method for Detection and Quantification of CAO1-Edited Rice
por: Zhang, Hongwen, et al.
Publicado: (2021)

Perspectives on the Genomics of HSP Beyond Mendelian Inheritance
por: Bis-Brewer, Dana M., et al.
Publicado: (2018)

Progress in Inherited Retinal Disease Drug Discovery and Development: A Foundation’s Perspective
por: Yerxa, Benjamin
Publicado: (2018)

Site-Specific Integration of Exogenous Genes Using Genome Editing Technologies in Zebrafish
por: Kawahara, Atsuo, et al.
Publicado: (2016)

Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy
por: Sanjurjo-Soriano, Carla, et al.
Publicado: (2018)

Patterns of genomic site inheritance in HIV-1M inter-subtype recombinants delineate the most likely genomic sites of subtype-specific adaptation
por: Tongo, Marcel, et al.
Publicado: (2018)

Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes
por: Rogers, Geoffrey L., et al.
Publicado: (2021)

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?
por: Bellingrath, Julia-Sophia, et al.
Publicado: (2023)

Single-Base Pair Genome Editing in Human Cells by Using Site-Specific Endonucleases
por: Ochiai, Hiroshi
Publicado: (2015)

A non-viral genome editing platform for site-specific insertion of large transgenes
por: Chaudhari, Namrata, et al.
Publicado: (2020)

Perspectives in Genome-Editing Techniques for Livestock
por: Popova, Julia, et al.
Publicado: (2023)

Chondrocyte-specific genomic editing enabled by hybrid exosomes for osteoarthritis treatment
por: Liang, Yujie, et al.
Publicado: (2022)

Genome editing in the treatment of ocular diseases
por: Choi, Elliot H., et al.
Publicado: (2023)

Efficient and Safe Editing of Porcine Endogenous Retrovirus Genomes by Multiple-Site Base-Editing Editor
por: Zheng, Shuwen, et al.
Publicado: (2022)

New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies
por: Pulman, Juliette, et al.
Publicado: (2022)

Progress in Genome Editing Technology and Its Application in Plants
por: Zhang, Kai, et al.
Publicado: (2017)

The CRISPR/Cas9 System and the Possibility of Genomic Edition for Cardiology
por: Arend, Marcela Corso, et al.
Publicado: (2017)

Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs
por: Yang, Fei, et al.
Publicado: (2023)

Genome-wide profiling of RNA editing sites in sheep
por: Zhang, Yuanyuan, et al.
Publicado: (2019)

Obstacles to Early Diagnosis and Treatment of Inherited von Willebrand Disease: Current Perspectives
por: Castaman, Giancarlo, et al.
Publicado: (2021)

New Possibilities on the Horizon: Genome Editing Makes the Whole Genome Accessible for Changes
por: Kawall, Katharina
Publicado: (2019)

Impaired histone inheritance promotes tumor progression
por: Tian, Congcong, et al.
Publicado: (2023)

Perspectives of Genome-Editing Technologies for HIV Therapy
por: Ebina, Hirotaka, et al.
Publicado: (2016)

Identification of genomic sites for CRISPR/Cas9-based genome editing in the Vitis vinifera genome
por: Wang, Yi, et al.
Publicado: (2016)

Progress in genomics according to bingo: 2013 edition
por: Karczewski, Konrad J
Publicado: (2013)

Genome editing: progress and challenges for medical applications
por: Carroll, Dana
Publicado: (2016)

Cannot write session to /tmp/vufind_sessions/sess_9bjd3jkp0lkikfor583bg0lums