Ethical and regulatory aspects of genome editing

Blood

Volumn 127, Issue 21, 2016, Pages 2553-2560

  Kohn, Donald B ^a,b   Porteus, Matthew H ^c   Scharenberg, Andrew M ^d,e  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c STANFORD UNIVERSITY   (United States)

^d UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^e SEATTLE CHILDREN S RESEARCH INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CRISPR ASSOCIATED PROTEIN; ENDONUCLEASE; NUCLEASE; ZINC FINGER NUCLEASE;

BIOETHICS; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; DNA END JOINING REPAIR; DNA MODIFICATION; DNA REPAIR; EX VIVO STUDY; GENE TECHNOLOGY; GENETIC PROCEDURES; GENOME EDITING; GERM LINE; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN GENOME; PRIORITY JOURNAL; REVIEW; RISK ASSESSMENT; SOMATIC CELL; ANIMAL; ETHICS; GENE EDITING; LEGISLATION AND JURISPRUDENCE; PROCEDURES; TARGETED GENE REPAIR;

ANIMALS; BIOETHICAL ISSUES; GENE EDITING; HUMANS; TARGETED GENE REPAIR;

EID: 84974622788     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-01-678136     Document Type: Review

References (35)

1. Khan IF, Hirata RK, Russell DW. AAV-mediated gene targeting methods for human cells. Nat Protoc. 2011;6(4):482-501.
2. Boissel S, Jarjour J, Astrakhan A, et al. megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering. Nucleic Acids Res. 2014;42(4):2591-2601.
3. Scharenberg AM, Duchateau P, Smith J. Genome engineering with TAL-effector nucleases and alternative modular nuclease technologies. Curr Gene Ther. 2013;13(4):291-303.
4. Porteus MH. Genome editing of the blood: opportunities and challenges. Curr Stem Cell Rep. 2015;1(1):23-30.
5. Corrigan-Curay J, O'Reilly M, Kohn DB, et al. Genome editing technologies: defining a path to clinic. Mol Ther. 2015;23(5):796-806.
6. Rasko JEJ, O'Sullivan GM, Ankeny RA. Is inheritable genetic modification the new dividing line? In: Rasko JEJ, O'Sullivan GM, Ankeny RA, eds. The Ethics of Inheritable Genetic Modification: A Dividing Line? Cambridge, United Kingdom: Cambridge University Press; 2006:1-16.
7. Friedmann T, Jonlin EC, King NM, et al. ASGCT and JSGT joint position statement on human genomic editing. Mol Ther. 2015;23(8):1282.
8. Porteus MH, Dann CT. Genome editing of the germline: broadening the discussion. Mol Ther. 2015;23(6):980-982.
9. Araki M, Ishii T. International regulatory landscape and integration of corrective genome editing into in vitro fertilization. Reprod Biol Endocrinol. 2014;12:108.
10. Evitt NH, Mascharak S, Altman RB. Human germline CRISPR-Cas modification: toward a regulatory framework. Am J Bioeth. 2015;15(12):25-29.
11. Krishan K, Kanchan T, Singh B. Human genome editing and ethical considerations. Sci Eng Ethics. 2016;22(2):597-599.
12. The National Academies of Sciences, Engineering, and Medicine. Academies' consensus study on human gene editing begins; first data-gathering meeting Feb. 11-12, 2016. http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID512082015. Accessed January 18, 2016.
13. Arruda VR, Fields PA, Milner R, et al. Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males. Mol Ther. 2001;4(6):586-592.
14. Schuettrumpf J, Liu JH, Couto LB, et al. Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial. [published correction appears in Mol Ther. 2006;14(6):893]. Mol Ther. 2006;13(6):1064-1073.
15. Sarkar R, Xiao W, Kazazian HH Jr. A single adeno-associated virus (AAV)-murine factor VIII vector partially corrects the hemophilia A phenotype. J Thromb Haemost. 2003;1(2):220-226.
16. National Institutes of Health. National Institutes of Health guidelines on human stem cell research. http://stemcells.nih.gov/policy/pages/2009guidelines.aspx. Accessed January 18, 2016.
17. Vogel G. U.K. researcher receives permission to edit genes in human embryos. http://www. sciencemag.org/news/2016/02/uk-researcher-receives-permission-edit-genes-human-embryos. Accessed January 18, 2016.
18. Tebas P, Stein D, Tang WW, et al. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med. 2014;370(10):901-910.
19. Qasim W, Amrolia PJ, Samarasinghe S, et al. First clinical application of Talen engineered universal CAR19 T cells in B-ALL [abstract]. Blood. 2015;126(23). Abstract 2046.
20. Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013;341(6148):1233158.
21. Hendel A, Kildebeck EJ, Fine EJ, et al. Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing. Cell Reports. 2014;7(1):293-305.
22. Miller DG, Petek LM, Russell DW. Adeno-associated virus vectors integrate at chromosome breakage sites. Nat Genet. 2004;36(7):767-773.
23. Gabriel R, Lombardo A, Arens A, et al. An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol. 2011;29(9):816-823.
24. Mátrai J, Cantore A, Bartholomae CC, et al. Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigenspecific tolerance in mice with low genotoxic risk. Hepatology. 2011;53(5):1696-1707.
25. Tsai SQ, Zheng Z, Nguyen NT, et al. GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases. Nat Biotechnol. 2015;33(2):187-197.
26. Kim D, Bae S, Park J, et al. Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells. Nat Methods. 2015;12(3):237-243.
27. Crosetto N, Mitra A, Silva MJ, et al. Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing. Nat Methods. 2013;10(4):361-365.
28. Frock RL, Hu J, Meyers RM, Ho YJ, Kii E, Alt FW. Genome-wide detection of DNA double-stranded breaks induced by engineered nucleases. Nat Biotechnol. 2015;33(2):179-186.
29. Pruett-Miller SM, Connelly JP, Maeder ML, Joung JK, Porteus MH. Comparison of zinc finger nucleases for use in gene targeting in mammalian cells. Mol Ther. 2008;16(4):707-717.
30. Miller JC, Holmes MC, Wang J, et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol. 2007;25(7):778-785.
31. Mussolino C, Alzubi J, Fine EJ, et al. TALENs facilitate targeted genome editing in human cells with high specificity and low cytotoxicity. Nucleic Acids Res. 2014;42(10):6762-6773.
32. Porter SN, Baker LC, Mittelman D, Porteus MH. Lentiviral and targeted cellular barcoding reveals ongoing clonal dynamics of cell lines in vitro and in vivo. Genome Biol. 2014;15(5):R75.
33. Modlich U, Bohne J, Schmidt M, et al. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood. 2006;108(8):2545-2553.
34. Poirot L, Philip B, Schiffer-Mannioui C, et al. Multiplex genome-edited T-cell manufacturing platform for "off-the-shelf" adoptive T-cell immunotherapies. Cancer Res. 2015;75(18):3853-3864.
35. Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371(21):1994-2004.