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
Citations
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Book ChapterDOI
Genetic Engineering in Stem Cell Biomanufacturing
Xiaoping Bao,Sean P. Palecek +1 more
TL;DR: This chapter briefly summarizes a variety of genetic manipulation strategies used to generate useful hPSC lines, and reviews their advantages and shortcomings to provide insights on suitable approaches for stem cell biomanufacturing applications.
Journal ArticleDOI
Fludarabine modulates expression of type VII collagen during haematopoietic stem cell transplantation for recessive dystrophic epidermolysis bullosa.
TL;DR: In this article, the effects of fludarabine on the production of type VII collagen in RDEB fibroblasts were investigated using reversetranscription polymerase chain reaction (RPCR) and immunoblotting.
Dissertation
Generation and characterization of novel adenoviral vectors for hybrid nuclease-mediated gene targeting
TL;DR: Tese de mestrado [versao publica].
Molecular and Genetic Medicine
TL;DR: It is believed that with further improvements these viral vectors for TALEN delivery will play an emerging role in bioengineering and for establishing novel therapeutic concepts.
DissertationDOI
Synthetic Regulation of Eukaryotic Gene Expression by Noncoding RNA
TL;DR: This work extends the use of synthetic self-cleaving ribozyme switches to detect changes in input protein levels and convey that information into programmed gene expression in eukaryotic cells, and develops a modular genetic amplifier for near-digital control of eUKaryotic gene expression.
References
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Journal ArticleDOI
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.