TALEN-based Gene Correction for Epidermolysis Bullosa
Mark J. Osborn,Colby G. Starker,Amber N. McElroy,Beau R. Webber,Megan J. Riddle,Lily Xia,Anthony P. DeFeo,Richard Gabriel,Manfred Schmidt,Christof von Kalle,Daniel F. Carlson,Morgan L. Maeder,J. Keith Joung,John E. Wagner,Daniel F. Voytas,Bruce R. Blazar,Jakub Tolar +16 more
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TLDR
This study provides proof-of-concept for TALen-mediated in situ correction of an endogenous patient-specific gene mutation and used an unbiased screen for comprehensive TALEN target mapping that will cooperatively facilitate translational application.About:
This article is published in Molecular Therapy.The article was published on 2013-06-01 and is currently open access. It has received 286 citations till now. The article focuses on the topics: Transcription activator-like effector nuclease & Gene mutation.read more
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From Gene Targeting to Genome Editing: Transgenic animals applications and beyond.
Mauricio Rocha-Martins,Gabriel R. Cavalheiro,Gabriel E. Matos-Rodrigues,Rodrigo A. P. Martins +3 more
TL;DR: A brief historical perspective of genome modification methods is presented, focusing on transgenic mice models, and how new techniques were discovered and improved, present the paradigm shifts and discuss their limitations and applications for biomedical research as well as possible future directions.
Journal ArticleDOI
Enhancing the specificity of recombinase-mediated genome engineering through dimer interface redesign.
TL;DR: The results provide a general means for improving hybrid recombinase specificity by protein engineering and illustrate the potential of these enzymes for basic research and therapeutic applications.
Journal ArticleDOI
Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector
Joanna Jacków,Matthias Titeux,Matthias Titeux,Soizic Portier,Soizic Portier,Soëli Charbonnier,Soëli Charbonnier,Clarisse Ganier,Clarisse Ganier,Sonia Gaucher,Alain Hovnanian,Alain Hovnanian +11 more
TL;DR: These data show the efficacy and safety of gene-corrected fibroblast therapy using a self-inactivating vector that has now been good manufacturing grade-certified and pave the way for clinical translation to treat nonhealing wounds in RDEB patients.
Journal ArticleDOI
Genome-wide Specificity of Highly Efficient TALENs and CRISPR/Cas9 for T Cell Receptor Modification
Friederike Knipping,Mark J. Osborn,Karl Petri,Jakub Tolar,Hanno Glimm,Christof von Kalle,Manfred Schmidt,Richard Gabriel +7 more
TL;DR: To knock out endogenous TCR expression, 12 transcription activator-like effector nucleases (TALENs) and five guide RNAs (gRNAs) from the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system are assembled and analysis of TALEN and CRISPR/Cas9 specificity is revealed, indicating a high level of specificity.
Journal ArticleDOI
Epidermolysis bullosa: Advances in research and treatment
TL;DR: This review discusses molecular procedures currently under investigation at the EB House Austria, a designated Centre of Expertise implemented in the European Reference Network for Rare and Undiagnosed Skin Diseases, and highlights the recent advancements of gene therapy development for EB.
References
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Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,Le Cong,F. Ann Ran,F. Ann Ran,David M. Cox,David M. Cox,Shuailiang Lin,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +15 more
TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,F. A. Ran,David Benjamin Turitz Cox,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +10 more
TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
Journal ArticleDOI
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali,Luhan Yang,Kevin M. Esvelt,John Aach,Marc Güell,James E. DiCarlo,Julie E. Norville,George M. Church,George M. Church +8 more
TL;DR: The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
Journal ArticleDOI
Enzymatic assembly of DNA molecules up to several hundred kilobases
Daniel G. Gibson,Lei Young,Ray-Yuan Chuang,J. Craig Venter,Clyde A. Hutchison,Hamilton O. Smith +5 more
TL;DR: An isothermal, single-reaction method for assembling multiple overlapping DNA molecules by the concerted action of a 5′ exonuclease, a DNA polymerase and a DNA ligase is described.
Journal ArticleDOI
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Salima Hacein-Bey-Abina,C von Kalle,C von Kalle,Manfred Schmidt,Matthew P. McCormack,NM Wulffraat,Philippe Leboulch,Annick Lim,Cameron S. Osborne,R. Pawliuk,Estelle Morillon,R. Sorensen,A. Forster,Peter Fraser,Jeffrey I. Cohen,G de Saint Basile,Ian E. Alexander,Uwe Wintergerst,Thierry Frebourg,Alain Aurias,Dominique Stoppa-Lyonnet,Serge Romana,I. Radford-Weiss,Fabian Gross,Françoise Valensi,Eric Delabesse,Elizabeth Macintyre,F. Sigaux,Jean Soulier,L. E. Leiva,Manuela Wissler,Claudia Prinz,Terence H. Rabbitts,F. Le Deist,Alain Fischer,Marina Cavazzana-Calvo +35 more
TL;DR: Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.