Recent Advances in CRISPR/Cas9 Delivery Approaches for Therapeutic Gene Editing of Stem Cells

[1] Guha TK(2017)Programmable genome editing tools and their regulation for efficient genome engineering Computational and Structural Biotechnology Journal 15 146-160

[2] Wai A(2014)Genome engineering with targetable nucleases Annual Review of Biochemistry 83 409-439

[3] Hausner G(2020)Applications of genome editing technology in the targeted therapy of human diseases: Mechanisms, advances and prospects Signal Transduction and Targeted Therapy 5 1-23

[4] Carroll D(2013)Multiplex genome engineering using CRISPR/Cas systems Science 339 819-823

[5] Li H(2013)Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients Cell Stem Cell 13 653-658

[6] Yang Y(2015)Functional gene correction for cystic fibrosis in lung epithelial cells generated from patient iPSCs Cell Reports 12 1385-1390

[7] Hong W(2016)CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells Nature 539 384-389

[8] Huang M(2021)CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia New England Journal of Medicine 384 252-260

[9] Wu M(2016)Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9 Human Molecular Genetics 25 4566-4576

[10] Zhao X(2017)CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo Molecular Therapy 25 12-23

[1] Guha TK(2017)Programmable genome editing tools and their regulation for efficient genome engineering Computational and Structural Biotechnology Journal 15 146-160

[2] Wai A(2014)Genome engineering with targetable nucleases Annual Review of Biochemistry 83 409-439

[3] Hausner G(2020)Applications of genome editing technology in the targeted therapy of human diseases: Mechanisms, advances and prospects Signal Transduction and Targeted Therapy 5 1-23

[4] Carroll D(2013)Multiplex genome engineering using CRISPR/Cas systems Science 339 819-823

[5] Li H(2013)Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients Cell Stem Cell 13 653-658

[6] Yang Y(2015)Functional gene correction for cystic fibrosis in lung epithelial cells generated from patient iPSCs Cell Reports 12 1385-1390

[7] Hong W(2016)CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells Nature 539 384-389

[8] Huang M(2021)CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia New England Journal of Medicine 384 252-260

[9] Wu M(2016)Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9 Human Molecular Genetics 25 4566-4576

[10] Zhao X(2017)CRISPR/Cas9 editing of the mutant huntingtin allele in vitro and in vivo Molecular Therapy 25 12-23