Navigation Links
A new genome editing method brings the possibility of gene therapies closer to reality
Date:7/11/2014

July 3, 2014, Shenzhen, China Researchers from Salk Institute for Biological Studies, BGI, and other institutes for the first time evaluated the safety and reliability of the existing targeted gene correction technologies, and successfully developed a new method, TALEN-HDAdV, which could significantly increased gene-correction efficiency in human induced pluripotent stem cell (hiPSC). This study published online in Cell Stell Cell provides an important theoretical foundation for stem cell-based gene therapy.

The combination of stem cells and targeted genome editing technology provides a powerful tool to model human diseases and develop potential cell replacement therapy. Although the utility of genome editing has been extensively documented, but the impact of these technologies on mutational load at the whole-genome level remains unclear.

In the study, researchers performed whole-genome sequencing to evaluate the mutational load at single-base resolution in individual gene-corrected hiPSC clones in three different disease models, including Hutchinson-Gilford progeria syndrome (HGPS), sickle cell disease (SCD), and Parkinson's disease (PD).

They evaluated the efficiencies of gene-targeting and gene-correction at the haemoglobin gene HBB locus with TALEN, HDAdV, CRISPR/CAS9 nuclease, and found the TALENs, HDAdVs and CRISPR/CAS9 mediated gene-correction methods have a similar efficiency at the gene HBB locus. In addition, the results of deep whole-genome sequencing indicated that TALEN and HDAdV could keep the patient's genome integrated at a maximum level, proving the safety and reliability of these methods.

Through integrating the advantages of TALEN- and HDAdV-mediated genome editing, researchers developed a new TALEN-HDAdV hybrid vector (talHDAdV), which can significantly increase the gene-correction efficiency in hiPSCs. Almost all the genetic mutations at the gene HBB locus can be detected by telHDAdV, which allows this new developed technology can be applied into the gene repair of different kinds of hemoglobin diseases such as SCD and Thalassemia.


'/>"/>

Contact: Jia Liu
liujia@genomics.cn
BGI Shenzhen
Source:Eurekalert

Related biology news :

1. Leading evolutionary scientist to discuss how genome of bacteria has evolved
2. Darwin in the genome
3. Analysis of stickleback genome sequence catches evolution in action
4. Athletic frogs have faster-changing genomes
5. PNAS: Precise molecular surgery in the plant genome
6. BGI and Aspera collaborate on high-speed data exchange to advance genome research
7. Researchers announce GenomeSpace environment to connect genomic tools
8. UC Santa Cruz builds national data center for cancer genome research
9. BGI reports the completed sequence of foxtail millet genome
10. Relative reference: Foxtail millet offers clues for assembling the switchgrass genome
11. Maps of Miscanthus genome offer insight into grass evolution
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/3/2016)... --> --> Fourth quarter 2015:   ... fourth quarter of 2014. Gross margin was 46% (32). ... Earnings per share increased to SEK 6.39 (loss: 0.49). Cash ... --> --> January to December ... 1,142% compared with 2014. Gross margin was 43% (31). ...
(Date:2/3/2016)... http://www.researchandmarkets.com/research/d8zjcd/emotion_detection ) ... "Emotion Detection and Recognition Market by Technology ... Tools (Facial Expression, Voice Recognition and Others), ... Global forecast to 2020" report to ... ) has announced the addition of the ...
(Date:2/2/2016)... 2, 2016 Checkpoint Inhibitors for Cancer ... Are you interested in the future of ... checkpoint inhibitors. Visiongain,s report gives those predictions to ... national level. Avoid falling behind in data ... and revenues those emerging cancer therapies can achieve. ...
Breaking Biology News(10 mins):
(Date:2/8/2016)... 8, 2016  NanoViricides, Inc. (NYSE MKT: NNVC) (the "Company"), a ... Seymour , MD, MPH, will present information about the company,s programs ... in New York City . ... Windsor Room at 5:30PM EST. Registered attendees can request a one ... City . --> New York City ...
(Date:2/6/2016)... ... 2016 , ... The Center for Excellence in Education (CEE) will sponsor a ... Wednesday February 10, 2016. This Bite of Science session, hosted by the Smithsonian-Mason ... 1500 Remount Road in Front Royal, VA from 5:00 p.m. to 8:00 p.m. The ...
(Date:2/5/2016)... 5, 2016 On Thursday, February 11, ... for community, health and disaster services, and the ... to enhance care coordination and service delivery for the ... need and to better connect service providers to the ... San Diego has handled more than ...
(Date:2/4/2016)... LEXINGTON, Massachusetts , February 4, 2016 - New ... --> - New FDA action date of July ... date of July 22, 2016   - ... the U.S. in the past decade indicated for the treatment of signs and ... Lifitegrast has the potential to be the only product approved in the ...
Breaking Biology Technology: