Literature DB >> 22129804

Safe harbours for the integration of new DNA in the human genome.

Michel Sadelain1, Eirini P Papapetrou, Frederic D Bushman.   

Abstract

Interactions between newly integrated DNA and the host genome limit the reliability and safety of transgene integration for therapeutic cell engineering and other applications. Although targeted gene delivery has made considerable progress, the question of where to insert foreign sequences in the human genome to maximize safety and efficacy has received little attention. In this Opinion article, we discuss 'genomic safe harbours' - chromosomal locations where therapeutic transgenes can integrate and function in a predictable manner without perturbing endogenous gene activity and promoting cancer.

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Year:  2011        PMID： 22129804     DOI： 10.1038/nrc3179

Source DB:  PubMed          Journal:  Nat Rev Cancer        ISSN： 1474-175X            Impact factor:   60.716


  85 in total

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Journal:  Blood       Date:  2003-07-24       Impact factor: 22.113

Review 2.  Genetic manipulation of genomes with rare-cutting endonucleases.

Authors:  M Jasin
Journal:  Trends Genet       Date:  1996-06       Impact factor: 11.639

3.  Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells.

Authors:  B P Zambrowicz; A Imamoto; S Fiering; L A Herzenberg; W G Kerr; P Soriano
Journal:  Proc Natl Acad Sci U S A       Date:  1997-04-15       Impact factor: 11.205

4.  Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity.

Authors:  Ute Modlich; Jens Bohne; Manfred Schmidt; Christof von Kalle; Sabine Knöss; Axel Schambach; Christopher Baum
Journal:  Blood       Date:  2006-07-06       Impact factor: 22.113

5.  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

6.  Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

Authors:  Marion G Ott; Manfred Schmidt; Kerstin Schwarzwaelder; Stefan Stein; Ulrich Siler; Ulrike Koehl; Hanno Glimm; Klaus Kühlcke; Andrea Schilz; Hana Kunkel; Sonja Naundorf; Andrea Brinkmann; Annette Deichmann; Marlene Fischer; Claudia Ball; Ingo Pilz; Cynthia Dunbar; Yang Du; Nancy A Jenkins; Neal G Copeland; Ursula Lüthi; Moustapha Hassan; Adrian J Thrasher; Dieter Hoelzer; Christof von Kalle; Reinhard Seger; Manuel Grez
Journal:  Nat Med       Date:  2006-04-02       Impact factor: 53.440

7.  Increased complexity of wild-type adeno-associated virus-chromosomal junctions as determined by analysis of unselected cellular genomes.

Authors:  Horace R Drew; Linda J Lockett; Gerald W Both
Journal:  J Gen Virol       Date:  2007-06       Impact factor: 3.891

8.  Transcription start regions in the human genome are favored targets for MLV integration.

Authors:  Xiaolin Wu; Yuan Li; Bruce Crise; Shawn M Burgess
Journal:  Science       Date:  2003-06-13       Impact factor: 47.728

9.  Efficient recombinase-mediated cassette exchange at the AAVS1 locus in human embryonic stem cells using baculoviral vectors.

Authors:  Chrishan J A Ramachandra; Mohammad Shahbazi; Timothy W X Kwang; Yukti Choudhury; Xiao Ying Bak; Jing Yang; Shu Wang
Journal:  Nucleic Acids Res       Date:  2011-06-17       Impact factor: 16.971

10.  Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting.

Authors:  Tomas Cermak; Erin L Doyle; Michelle Christian; Li Wang; Yong Zhang; Clarice Schmidt; Joshua A Baller; Nikunj V Somia; Adam J Bogdanove; Daniel F Voytas
Journal:  Nucleic Acids Res       Date:  2011-04-14       Impact factor: 16.971
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  186 in total

Review 1.  Hematopoietic stem cell engineering at a crossroads.

Authors:  Isabelle Rivière; Cynthia E Dunbar; Michel Sadelain
Journal:  Blood       Date:  2011-11-17       Impact factor: 22.113

Review 2.  Current Progress in Therapeutic Gene Editing for Monogenic Diseases.

Authors:  Versha Prakash; Marc Moore; Rafael J Yáñez-Muñoz
Journal:  Mol Ther       Date:  2016-01-14       Impact factor: 11.454

3.  Identification of hematopoietic-specific regulatory elements from the CD45 gene and use for lentiviral tracking of transplanted cells.

Authors:  Khanh L Duong; Satyabrata Das; Shuyang Yu; Jennifer Y Barr; Snehalata Jena; Eunmi Kim; Nicolas Zavazava; John D Colgan; Hai-Hui Xue; Dana N Levasseur
Journal:  Exp Hematol       Date:  2014-05-20       Impact factor: 3.084

4.  Generation of an Induced Pluripotent Stem Cell Line with the Constitutive EGFP Reporter.

Authors:  Kiel T Butterfield; Patrick S McGrath; Chann Makara Han; Igor Kogut; Ganna Bilousova
Journal:  Methods Mol Biol       Date:  2020

5.  RNA-guided DNA insertion with CRISPR-associated transposases.

Authors:  Jonathan Strecker; Alim Ladha; Zachary Gardner; Jonathan L Schmid-Burgk; Kira S Makarova; Eugene V Koonin; Feng Zhang
Journal:  Science       Date:  2019-06-06       Impact factor: 47.728

6.  Genome editing technologies: defining a path to clinic.

Authors:  Jacqueline Corrigan-Curay; Marina O'Reilly; Donald B Kohn; Paula M Cannon; Gang Bao; Frederic D Bushman; Dana Carroll; Toni Cathomen; J Keith Joung; David Roth; Michel Sadelain; Andrew M Scharenberg; Christof von Kalle; Feng Zhang; Robert Jambou; Eugene Rosenthal; Morad Hassani; Aparna Singh; Matthew H Porteus
Journal:  Mol Ther       Date:  2015-05       Impact factor: 11.454

7.  Hitting the target without pulling the trigger.

Authors:  Thierry VandenDriessche; Marinee K Chuah
Journal:  Mol Ther       Date:  2015-01       Impact factor: 11.454

8.  Generation of hypoimmunogenic human pluripotent stem cells.

Authors:  Xiao Han; Mengning Wang; Songwei Duan; Paul J Franco; Jennifer Hyoje-Ryu Kenty; Preston Hedrick; Yulei Xia; Alana Allen; Leonardo M R Ferreira; Jack L Strominger; Douglas A Melton; Torsten B Meissner; Chad A Cowan
Journal:  Proc Natl Acad Sci U S A       Date:  2019-04-30       Impact factor: 11.205

Review 9.  Delivery technologies for genome editing.

Authors:  Hao Yin; Kevin J Kauffman; Daniel G Anderson
Journal:  Nat Rev Drug Discov       Date:  2017-03-24       Impact factor: 84.694

10.  Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia.

Authors:  Shubhranshu Debnath; Pekka Jaako; Kavitha Siva; Michael Rothe; Jun Chen; Maria Dahl; H Bobby Gaspar; Johan Flygare; Axel Schambach; Stefan Karlsson
Journal:  Mol Ther       Date:  2017-04-20       Impact factor: 11.454
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