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
Reads0
Chats0
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
More filters
Dissertation
Engineering High-Precision CRISPR-Cas9 Nuclease and Base Editor Technologies
TL;DR: This work focuses on engineering CRISPR-Cas9 RNA-guided nucleases and base editor technologies to enhance their precision and genome-wide specificities, thus enabling researchers to more effectively use these tools to study fundamental biological principles or to translate these technologies into clinical settings.
DNA Scissors—TALEN and CRISPR/Cas
Ni Peiling,Liu Chang,Chen Lingyi +2 more
TL;DR: The recent progress and application of genome editing technology, with emphasis on two sequence-specific nucleases — TALEN and CRISPR/Cas are summarized.
Journal ArticleDOI
Dynamics and Emerging Trends in Genodermatology: A Scientometric Analysis
Diez años desde el descubrimiento de las células IPS: estado actual de su aplicación clínica
TL;DR: On the 10-year anniversary of the discovery of induced pluripotent stem cells, the main results from their various fields of application, the obstacles encountered during experimentation and the potential applications in clinical practice are reviewed.
Journal ArticleDOI
Genome-editing technologies and patent landscape overview.
TL;DR: This review compares key technologies of genome-editing zinc finger nucleases, transcriptional activator-like effector nucleases and CRISPR, with a focus on the race to acquire lucrative intellectual property rights, the currentCRISPR patent dispute and potential repercussions on innovation and the adoption of this promising technology by the medical community.
References
More filters
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.