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)... , Feb. 3, 2016 ... the addition of the "Emotion Detection ... Machine Learning, and Others), Software Tools (Facial ... Areas, End Users,and Regions - Global forecast ... --> http://www.researchandmarkets.com/research/d8zjcd/emotion_detection ) has ...
(Date:2/2/2016)... Feb. 2, 2016 Technology Enhancements Accelerate Growth of ... of the digital and computed radiography markets in ... and Indonesia (TIM). It provides ... size, as well as regional market drivers and restraints. ... market penetration and market attractiveness, both for digital and ...
(Date:2/1/2016)... , Feb. 1, 2016  Today, the first ... (AHA) announced plans to develop a first of its ... power of IBM Watson. In the first application of ... IBM (NYSE: IBM ), and Welltok will create ... health assessments with cognitive analytics, delivered on Welltok,s health ...
Breaking Biology News(10 mins):
(Date:2/10/2016)... Early-career researchers from Indonesia , ... Uganda and Yemen honored ... Indonesia , Nepal , ... are being honored for their accomplishments in nutrition, psychiatry, biotechnology, ... young women scientists who are pursuing careers in agriculture, biology and medicine ...
(Date:2/10/2016)...  IsoRay, Inc. (NYSE MKT: ISR), a medical technology ... applications for the treatment of prostate, brain, lung, head ... results for the second quarter and six months of ... --> --> Revenue was ... which ended December 31, 2015, a 12% increase compared ...
(Date:2/10/2016)... ... ... LATHAM, NEW YORK... Marktech Optoelectronics will feature their new high-speed InGaAs ... Moscone Center from February 16-18, 2016, and at the healthcare-focused BiOS Expo on February ... standard packages feature a TO-46 metal can with active areas of 1.0mm and 1.5mm ...
(Date:2/10/2016)... (PRWEB) , ... February 10, 2016 , ... Cenna Bioscience ... for the treatment of Alzheimer’s disease, announced today it has been selected to present ... at the Breakers in Palm Beach, Florida. The purpose of the Forum is ...
Breaking Biology Technology: