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Literature DB >> 34619370

CRISPR-derived genome editing therapies: Progress from bench to bedside.

Holly A Rees¹, Alex C Minella¹, Cameron A Burnett², Alexis C Komor³, Nicole M Gaudelli⁴.

Abstract

The development of CRISPR-derived genome editing technologies has enabled the precise manipulation of DNA sequences within the human genome. In this review, we discuss the initial development and cellular mechanism of action of CRISPR nucleases and DNA base editors. We then describe factors that must be taken into consideration when developing these tools into therapeutic agents, including the potential for unintended and off-target edits when using these genome editing tools, and methods to characterize these types of edits. We finish by considering specific challenges associated with bringing a CRISPR-based therapy to the clinic, including manufacturing, regulatory oversight, and considerations for clinical trials that involve genome editing agents.

Entities: Chemical

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Year: 2021 PMID： 34619370 PMCID： PMC8572140 DOI： 10.1016/j.ymthe.2021.09.027

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

129 in total

1. Eukaryotic translation initiation is controlled by cooperativity effects within ternary complexes of 4E-BP1, eIF4E, and the mRNA 5' cap.

Authors: Anna Modrak-Wojcik; Michal Gorka; Katarzyna Niedzwiecka; Konrad Zdanowski; Joanna Zuberek; Anna Niedzwiecka; Ryszard Stolarski
Journal: FEBS Lett Date: 2013-11-06 Impact factor: 4.124

Review 2. A guide to genome engineering with programmable nucleases.

Authors: Hyongbum Kim; Jin-Soo Kim
Journal: Nat Rev Genet Date: 2014-04-02 Impact factor: 53.242

3. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

Authors: Akin Akinc; William Querbes; Soma De; June Qin; Maria Frank-Kamenetsky; K Narayanannair Jayaprakash; Muthusamy Jayaraman; Kallanthottathil G Rajeev; William L Cantley; J Robert Dorkin; James S Butler; Liuliang Qin; Timothy Racie; Andrew Sprague; Eugenio Fava; Anja Zeigerer; Michael J Hope; Marino Zerial; Dinah W Y Sah; Kevin Fitzgerald; Mark A Tracy; Muthiah Manoharan; Victor Koteliansky; Antonin de Fougerolles; Martin A Maier
Journal: Mol Ther Date: 2010-05-11 Impact factor: 11.454

4. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Authors: Jinkuk Kim; Chunguang Hu; Christelle Moufawad El Achkar; Lauren E Black; Julie Douville; Austin Larson; Mary K Pendergast; Sara F Goldkind; Eunjung A Lee; Ashley Kuniholm; Aubrie Soucy; Jai Vaze; Nandkishore R Belur; Kristina Fredriksen; Iva Stojkovska; Alla Tsytsykova; Myriam Armant; Renata L DiDonato; Jaejoon Choi; Laura Cornelissen; Luis M Pereira; Erika F Augustine; Casie A Genetti; Kira Dies; Brenda Barton; Lucinda Williams; Benjamin D Goodlett; Bobbie L Riley; Amy Pasternak; Emily R Berry; Kelly A Pflock; Stephen Chu; Chantal Reed; Kimberly Tyndall; Pankaj B Agrawal; Alan H Beggs; P Ellen Grant; David K Urion; Richard O Snyder; Susan E Waisbren; Annapurna Poduri; Peter J Park; Al Patterson; Alessandra Biffi; Joseph R Mazzulli; Olaf Bodamer; Charles B Berde; Timothy W Yu
Journal: N Engl J Med Date: 2019-10-09 Impact factor: 91.245

5. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

Authors: Salima Hacein-Bey-Abina; Alexandrine Garrigue; Gary P Wang; Jean Soulier; Annick Lim; Estelle Morillon; Emmanuelle Clappier; Laure Caccavelli; Eric Delabesse; Kheira Beldjord; Vahid Asnafi; Elizabeth MacIntyre; Liliane Dal Cortivo; Isabelle Radford; Nicole Brousse; François Sigaux; Despina Moshous; Julia Hauer; Arndt Borkhardt; Bernd H Belohradsky; Uwe Wintergerst; Maria C Velez; Lily Leiva; Ricardo Sorensen; Nicolas Wulffraat; Stéphane Blanche; Frederic D Bushman; Alain Fischer; Marina Cavazzana-Calvo
Journal: J Clin Invest Date: 2008-09 Impact factor: 14.808

6. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.

Authors: Shannon L Maude; Theodore W Laetsch; Jochen Buechner; Susana Rives; Michael Boyer; Henrique Bittencourt; Peter Bader; Michael R Verneris; Heather E Stefanski; Gary D Myers; Muna Qayed; Barbara De Moerloose; Hidefumi Hiramatsu; Krysta Schlis; Kara L Davis; Paul L Martin; Eneida R Nemecek; Gregory A Yanik; Christina Peters; Andre Baruchel; Nicolas Boissel; Francoise Mechinaud; Adriana Balduzzi; Joerg Krueger; Carl H June; Bruce L Levine; Patricia Wood; Tetiana Taran; Mimi Leung; Karen T Mueller; Yiyun Zhang; Kapildeb Sen; David Lebwohl; Michael A Pulsipher; Stephan A Grupp
Journal: N Engl J Med Date: 2018-02-01 Impact factor: 91.245

7. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

Authors: Morgan L Maeder; Michael Stefanidakis; Christopher J Wilson; Reshica Baral; Luis Alberto Barrera; George S Bounoutas; David Bumcrot; Hoson Chao; Dawn M Ciulla; Jennifer A DaSilva; Abhishek Dass; Vidya Dhanapal; Tim J Fennell; Ari E Friedland; Georgia Giannoukos; Sebastian W Gloskowski; Alexandra Glucksmann; Gregory M Gotta; Hariharan Jayaram; Scott J Haskett; Bei Hopkins; Joy E Horng; Shivangi Joshi; Eugenio Marco; Rina Mepani; Deepak Reyon; Terence Ta; Diana G Tabbaa; Steven J Samuelsson; Shen Shen; Maxwell N Skor; Pam Stetkiewicz; Tongyao Wang; Clifford Yudkoff; Vic E Myer; Charles F Albright; Haiyan Jiang
Journal: Nat Med Date: 2019-01-21 Impact factor: 53.440

CRISPR-derived genome editing therapies: Progress from bench to bedside.

1. Eukaryotic translation initiation is controlled by cooperativity effects within ternary complexes of 4E-BP1, eIF4E, and the mRNA 5' cap.

Review 2. A guide to genome engineering with programmable nucleases.

3. Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms.

4. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

5. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

6. Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.

7. Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10.

8. Genome-wide profiling of adenine base editor specificity by EndoV-seq.

9. Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit.

10. crRNA and tracrRNA guide Cas9-mediated DNA interference in Streptococcus thermophilus.

Review 1. Therapeutic in vivo delivery of gene editing agents.