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Advances in CRISPR/Cas systems-based cell and gene therapy.
Poddar, Arpita; Ahmady, Farah; Prithviraj, Prashanth; Luwor, Rodney B; Shukla, Ravi; Polash, Shakil Ahmed; Li, Haiyan; Ramakrishna, Suresh; Kannourakis, George; Jayachandran, Aparna.
Afiliación
  • Poddar A; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia; RMIT University, VIC, Australia.
  • Ahmady F; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia.
  • Prithviraj P; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia.
  • Luwor RB; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia; The University of Melbourne, Parkville, VIC, Australia; Huagene Institute, Kecheng Science and Technology Park, Pukou, Nanjing, P.R. China.
  • Shukla R; RMIT University, VIC, Australia.
  • Polash SA; RMIT University, VIC, Australia.
  • Li H; RMIT University, VIC, Australia.
  • Ramakrishna S; Hanyang University, Seoul, South Korea.
  • Kannourakis G; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia.
  • Jayachandran A; Fiona Elsey Cancer Research Institute, VIC, Australia; Federation University, VIC, Australia. Electronic address: aparna@fecri.org.au.
Prog Mol Biol Transl Sci ; 208: 161-183, 2024.
Article en En | MEDLINE | ID: mdl-39266181
ABSTRACT
Cell and gene therapy are innovative biomedical strategies aimed at addressing diseases at their genetic origins. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) systems have become a groundbreaking tool in cell and gene therapy, offering unprecedented precision and versatility in genome editing. This chapter explores the role of CRISPR in gene editing, tracing its historical development and discussing biomolecular formats such as plasmid, RNA, and protein-based approaches. Next, we discuss CRISPR delivery methods, including viral and non-viral vectors, followed by examining the various engineered CRISPR variants for their potential in gene therapy. Finally, we outline emerging clinical applications, highlighting the advancements in CRISPR for breakthrough medical treatments.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Sistemas CRISPR-Cas / Edición Génica Límite: Animals / Humans Idioma: En Revista: Prog Mol Biol Transl Sci Asunto de la revista: BIOLOGIA MOLECULAR Año: 2024 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Países Bajos
Texto completo
Añadir a Mi BVS
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XML
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Sistemas CRISPR-Cas / Edición Génica Límite: Animals / Humans Idioma: En Revista: Prog Mol Biol Transl Sci Asunto de la revista: BIOLOGIA MOLECULAR Año: 2024 Tipo del documento: Article País de afiliación: Australia Pais de publicación: Países Bajos