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Literature DB >> 26765770

Current Progress in Therapeutic Gene Editing for Monogenic Diseases.

Versha Prakash¹, Marc Moore¹, Rafael J Yáñez-Muñoz¹.

Abstract

Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and therapy. With regard to human gene therapy, nuclease-based gene editing has facilitated development of a broad range of therapeutic strategies based on both nonhomologous end joining and homology-dependent repair. This review discusses current progress in nuclease-based therapeutic applications for a subset of inherited monogenic diseases including cystic fibrosis, Duchenne muscular dystrophy, diseases of the bone marrow, and hemophilia and highlights associated challenges and future prospects.

Entities: Disease Species

Mesh：

Year: 2016 PMID： 26765770 PMCID： PMC4786935 DOI： 10.1038/mt.2016.5

Source DB: PubMed Journal: Mol Ther ISSN： 1525-0016 Impact factor: 11.454

114 in total

1. Functional engraftment of colon epithelium expanded in vitro from a single adult Lgr5⁺ stem cell.

Authors: Shiro Yui; Tetsuya Nakamura; Toshiro Sato; Yasuhiro Nemoto; Tomohiro Mizutani; Xiu Zheng; Shizuko Ichinose; Takashi Nagaishi; Ryuichi Okamoto; Kiichiro Tsuchiya; Hans Clevers; Mamoru Watanabe
Journal: Nat Med Date: 2012-03-11 Impact factor: 53.440

Review 2. Therapeutic gene targeting.

Authors: R J Yáñez; A C Porter
Journal: Gene Ther Date: 1998-02 Impact factor: 5.250

3. No factor left behind: generation of transgene-free induced pluripotent stem cells.

Authors: Mo Li; Juan Carlos Izpisua Belmonte
Journal: Am J Stem Cells Date: 2011-09-19

4. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors: Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal: Nature Date: 2010-09-16 Impact factor: 49.962

5. Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.

Authors: Ning Sun; Huimin Zhao
Journal: Biotechnol Bioeng Date: 2013-08-26 Impact factor: 4.530

6. A mechanism for the suppression of homologous recombination in G1 cells.

Authors: Alexandre Orthwein; Sylvie M Noordermeer; Marcus D Wilson; Sébastien Landry; Radoslav I Enchev; Alana Sherker; Meagan Munro; Jordan Pinder; Jayme Salsman; Graham Dellaire; Bing Xia; Matthias Peter; Daniel Durocher
Journal: Nature Date: 2015-12-09 Impact factor: 49.962

Review 7. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Authors: Annemieke Aartsma-Rus; Ivo Fokkema; Jan Verschuuren; Ieke Ginjaar; Judith van Deutekom; Gert-Jan van Ommen; Johan T den Dunnen
Journal: Hum Mutat Date: 2009-03 Impact factor: 4.878

8. Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

Authors: H Bobby Gaspar; Samantha Cooray; Kimberly C Gilmour; Kathryn L Parsley; Stuart Adams; Steven J Howe; Abdulaziz Al Ghonaium; Jinhua Bayford; Lucinda Brown; E Graham Davies; Christine Kinnon; Adrian J Thrasher
Journal: Sci Transl Med Date: 2011-08-24 Impact factor: 17.956

9. megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.

Authors: Sandrine Boissel; Jordan Jarjour; Alexander Astrakhan; Andrew Adey; Agnès Gouble; Philippe Duchateau; Jay Shendure; Barry L Stoddard; Michael T Certo; David Baker; Andrew M Scharenberg
Journal: Nucleic Acids Res Date: 2013-11-26 Impact factor: 16.971

10. Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Authors: Fei Xie; Lin Ye; Judy C Chang; Ashley I Beyer; Jiaming Wang; Marcus O Muench; Yuet Wai Kan
Journal: Genome Res Date: 2014-08-05 Impact factor: 9.043

31 in total

Review 1. Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities.

Authors: Ling Li; Shuo Hu; Xiaoyuan Chen
Journal: Biomaterials Date: 2018-04-18 Impact factor: 12.479

Review 2. Emptying the stores: lysosomal diseases and therapeutic strategies.

Authors: Frances M Platt
Journal: Nat Rev Drug Discov Date: 2017-11-17 Impact factor: 84.694

Review 3. Gene Therapy for Hemophilia.

Authors: Arthur W Nienhuis; Amit C Nathwani; Andrew M Davidoff
Journal: Mol Ther Date: 2017-04-11 Impact factor: 11.454

Current Progress in Therapeutic Gene Editing for Monogenic Diseases.

1. Functional engraftment of colon epithelium expanded in vitro from a single adult Lgr5⁺ stem cell.

Review 2. Therapeutic gene targeting.

3. No factor left behind: generation of transgene-free induced pluripotent stem cells.

4. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

5. Seamless correction of the sickle cell disease mutation of the HBB gene in human induced pluripotent stem cells using TALENs.

6. A mechanism for the suppression of homologous recombination in G1 cells.

Review 7. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

8. Long-term persistence of a polyclonal T cell repertoire after gene therapy for X-linked severe combined immunodeficiency.

9. megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.

10. Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac.

Review 1. Non-viral delivery systems for CRISPR/Cas9-based genome editing: Challenges and opportunities.

Review 2. Emptying the stores: lysosomal diseases and therapeutic strategies.

Review 3. Gene Therapy for Hemophilia.

4. The effect of polar headgroups and spacer length on the DNA transfection of cholesterol-based cationic lipids.

Review 5. Adeno-associated virus as a gene therapy vector: strategies to neutralize the neutralizing antibodies.

6. Kinetics of Nuclear Uptake and Site-Specific DNA Cleavage during CRISPR-Directed Gene Editing in Solid Tumor Cells.

Review 7. Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications.

Review 8. Therapeutic Genome Editing and In Vivo Delivery.

Review 9. CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells.

Review 10. In vivo methods for acute modulation of gene expression in the central nervous system.