Literature DB >> 27231391

Knock-in editing: it functionally corrects!

Matthew H Porteus1.   

Abstract

Mesh:

Year:  2016        PMID: 27231391      PMCID: PMC4882800          DOI: 10.1182/blood-2016-03-703181

Source DB:  PubMed          Journal:  Blood        ISSN: 0006-4971            Impact factor:   22.113


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  10 in total

1.  Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing.

Authors:  Ayal Hendel; Eric J Kildebeck; Eli J Fine; Joseph Clark; Niraj Punjya; Vittorio Sebastiano; Gang Bao; Matthew H Porteus
Journal:  Cell Rep       Date:  2014-03-27       Impact factor: 9.423

2.  Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome.

Authors:  Nicholas Hubbard; David Hagin; Karen Sommer; Yumei Song; Iram Khan; Courtnee Clough; Hans D Ochs; David J Rawlings; Andrew M Scharenberg; Troy R Torgerson
Journal:  Blood       Date:  2016-02-22       Impact factor: 22.113

3.  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

4.  Lentiviral and murine retroviral transduction of T cells for expression of human CD40 ligand.

Authors:  S C Barry; J Seppen; N Ramesh; J L Foster; K Seyama; H D Ochs; J V Garcia; W R Osborne
Journal:  Hum Gene Ther       Date:  2000-01-20       Impact factor: 5.695

5.  Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template.

Authors:  Blythe D Sather; Guillermo S Romano Ibarra; Karen Sommer; Gabrielle Curinga; Malika Hale; Iram F Khan; Swati Singh; Yumei Song; Kamila Gwiazda; Jaya Sahni; Jordan Jarjour; Alexander Astrakhan; Thor A Wagner; Andrew M Scharenberg; David J Rawlings
Journal:  Sci Transl Med       Date:  2015-09-30       Impact factor: 17.956

6.  Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

Authors:  Ayal Hendel; Rasmus O Bak; Joseph T Clark; Andrew B Kennedy; Daniel E Ryan; Subhadeep Roy; Israel Steinfeld; Benjamin D Lunstad; Robert J Kaiser; Alec B Wilkens; Rosa Bacchetta; Anya Tsalenko; Douglas Dellinger; Laurakay Bruhn; Matthew H Porteus
Journal:  Nat Biotechnol       Date:  2015-06-29       Impact factor: 54.908

Review 7.  Genome Editing: A New Approach to Human Therapeutics.

Authors:  Matthew Porteus
Journal:  Annu Rev Pharmacol Toxicol       Date:  2015-11-09       Impact factor: 13.820

8.  A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.

Authors:  Salima Hacein-Bey-Abina; Sung-Yun Pai; H Bobby Gaspar; Myriam Armant; Charles C Berry; Stephane Blanche; Jack Bleesing; Johanna Blondeau; Helen de Boer; Karen F Buckland; Laure Caccavelli; Guilhem Cros; Satiro De Oliveira; Karen S Fernández; Dongjing Guo; Chad E Harris; Gregory Hopkins; Leslie E Lehmann; Annick Lim; Wendy B London; Johannes C M van der Loo; Nirav Malani; Frances Male; Punam Malik; M Angélica Marinovic; Anne-Marie McNicol; Despina Moshous; Benedicte Neven; Matías Oleastro; Capucine Picard; Jerome Ritz; Christine Rivat; Axel Schambach; Kit L Shaw; Eric A Sherman; Leslie E Silberstein; Emmanuelle Six; Fabien Touzot; Alla Tsytsykova; Jinhua Xu-Bayford; Christopher Baum; Frederic D Bushman; Alain Fischer; Donald B Kohn; Alexandra H Filipovich; Luigi D Notarangelo; Marina Cavazzana; David A Williams; Adrian J Thrasher
Journal:  N Engl J Med       Date:  2014-10-09       Impact factor: 91.245

9.  Nuclease-mediated gene editing by homologous recombination of the human globin locus.

Authors:  Richard A Voit; Ayal Hendel; Shondra M Pruett-Miller; Matthew H Porteus
Journal:  Nucleic Acids Res       Date:  2013-10-23       Impact factor: 16.971

10.  Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Authors:  Alessandra Biffi; Eugenio Montini; Laura Lorioli; Martina Cesani; Francesca Fumagalli; Tiziana Plati; Cristina Baldoli; Sabata Martino; Andrea Calabria; Sabrina Canale; Fabrizio Benedicenti; Giuliana Vallanti; Luca Biasco; Simone Leo; Nabil Kabbara; Gianluigi Zanetti; William B Rizzo; Nalini A L Mehta; Maria Pia Cicalese; Miriam Casiraghi; Jaap J Boelens; Ubaldo Del Carro; David J Dow; Manfred Schmidt; Andrea Assanelli; Victor Neduva; Clelia Di Serio; Elia Stupka; Jason Gardner; Christof von Kalle; Claudio Bordignon; Fabio Ciceri; Attilio Rovelli; Maria Grazia Roncarolo; Alessandro Aiuti; Maria Sessa; Luigi Naldini
Journal:  Science       Date:  2013-07-11       Impact factor: 47.728

  10 in total
  1 in total

1.  CD34+ cells from dental pulp stem cells with a ZFN-mediated and homology-driven repair-mediated locus-specific knock-in of an artificial β-globin gene.

Authors:  S Chattong; O Ruangwattanasuk; W Yindeedej; A Setpakdee; K Manotham
Journal:  Gene Ther       Date:  2017-05-22       Impact factor: 5.250

  1 in total

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