[HTML][HTML] In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease

CH Lau, Y Suh - F1000Research, 2017 - ncbi.nlm.nih.gov
Adeno-associated virus (AAV) has shown promising therapeutic efficacy with a good safety
profile in a wide range of animal models and human clinical trials. With the advent of …

[HTML][HTML] A self-deleting AAV-CRISPR system for in vivo genome editing

A Li, CM Lee, AE Hurley, KE Jarrett, M De Giorgi… - … Therapy-Methods & …, 2019 - cell.com
Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR)
can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV …

A multifunctional AAV–CRISPR–Cas9 and its host response

WL Chew, M Tabebordbar, JKW Cheng, P Mali… - Nature …, 2016 - nature.com
CRISPR–Cas9 delivery by adeno-associated virus (AAV) holds promise for gene therapy
but faces critical barriers on account of its potential immunogenicity and limited payload …

[PDF][PDF] CRISPR-based therapeutic genome editing: strategies and in vivo delivery by AAV vectors

D Wang, F Zhang, G Gao - Cell, 2020 - cell.com
The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …

Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing

T Wei, Q Cheng, L Farbiak, DG Anderson, R Langer… - ACS …, 2020 - ACS Publications
CRISPR/Cas9-based genome editing has quickly emerged as a powerful breakthrough
technology for use in diverse settings across biomedical research and therapeutic …

[HTML][HTML] CRISPR-Cas9 for in vivo gene therapy: Promise and hurdles

WJ Dai, LY Zhu, ZY Yan, Y Xu, QL Wang… - Molecular Therapy-Nucleic …, 2016 - cell.com
Owing to its easy-to-use and multiplexing nature, the genome editing tool CRISPR-Cas9
(clustered regularly interspaced short palindromic repeats (CRISPR) associated nuclease 9) …

CRISPR/Cas system for genome editing: progress and prospects as a therapeutic tool

DK Sahel, A Mittal, D Chitkara - Journal of Pharmacology and Experimental …, 2019 - ASPET
CRISPR was first observed in 1987 in bacteria and archaea and was later confirmed as part
of bacterial adaptive immunity against the attacking phage. The CRISPR/Cas restriction …

Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities

L Li, S Hu, X Chen - Biomaterials, 2018 - Elsevier
In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas
(CRISPR-associated) genome editing systems have become one of the most robust …

Delivery aspects of CRISPR/Cas for in vivo genome editing

D Wilbie, J Walther, E Mastrobattista - Accounts of chemical …, 2019 - ACS Publications
Conspectus The discovery of CRISPR/Cas has revolutionized the field of genome editing.
CRIPSR/Cas components are part of the bacterial immune system and are able to induce …

[HTML][HTML] Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

M Khoshandam, H Soltaninejad, M Mousazadeh… - Genes & …, 2024 - Elsevier
CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …