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
'/>"/>

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

Page: 1 2

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:5/11/2015)... Calif. , May 11, 2015  Synaptics ... of human interface solutions, today announced the appointment ... President and Chief Financial Officer, reporting to ... replaces Synaptics, current Chief Financial Officer, Kathleen ... 2014. Mr. Ali brings extensive ...
(Date:5/11/2015)... 2015 Curemark LLC, a privately held drug ... new Phase III double blind, randomized, placebo-controlled clinical trial ... on all children ages 3-8 with Autism. Previously, Curemark ... double blinded clinical trial for CM-AT in children ages ... digestive enzyme chymotrypsin. This new trial will help determine ...
(Date:5/8/2015)... 2015 Synaptics Inc. (NASDAQ: SYNA ), the ... of the executive management team will present at the following ... and Telecom Conference Date: May 18, 2015 Time: ... MA Cowen and Company Technology, Media ... Location: The New York Palace Hotel, New York, ...
Breaking Biology News(10 mins):Synaptics Appoints Wajid Ali as Senior Vice President and Chief Financial Officer 2Curemark, LLC, Launches New Phase III Trial in Expanded Population of Children with Autism 2Synaptics to Present at Upcoming Investor Conferences 2
... a protein in the brain that plays a key role ... cell that supplies energy, supports cellular activity, and potentially wards ... today in the Journal of Cell Biology , may ... "Understanding the molecular machinery that helps distribute ...
... Australian and New Zealand researchers have accelerated research ... genes which will help to unravel the causes of ... findings will be published today in the prestigious journal ... of MS has remained a mystery. This discovery reveals ...
... College of Medicine have discovered how the mutated huntingtin gene ... Huntington,s disease. The report of their findings is available in ... to show that the mutated huntingtin gene activates a particular ... further, to show what effect activation of that enzyme has ...
Cached Biology News:Protein regulates movement of mitochondria in brain cells 2Protein regulates movement of mitochondria in brain cells 3Aussie and Kiwi researchers make double MS genetic discovery 2Huntington's disease deciphered 2Huntington's disease deciphered 3
(Date:5/21/2015)... Specialty Pharmacy Times has completed its ... BPA Worldwide as a business publication member. As ... for Specialty Pharmacy Times based on business/distribution, demographics ... member of BPA Worldwide, Specialty Pharmacy Times shows ... the most reliable, unequaled data,” said Chris Hennessy, ...
(Date:5/21/2015)... May 21, 2015 Imagine being ... - and those of others. Being able to read ... and read people to anticipate their thoughts and actions ... emotions and feelings to create unique abstract paintings and ... games. Get inside a first-person-shooter as it creates action, ...
(Date:5/21/2015)... 20, 2015 Research and Markets ... the "2015 Global Survey on Flow Cytometry ... The primary goal of this research is ... and reagents. Key information the survey seeks to ... flow cytometers, predominantly used applications for flow cytometers, ...
(Date:5/20/2015)... --Research and Markets ( http://www.researchandmarkets.com/research/x4b7kw/top_technologies ) has ... HW_Technical Insights" report to their offering. ... in the Health and Wellness sector and has identified ... impact in the year 2015. The research service provides ... and wellness technologies that are anticipated to have the ...
Breaking Biology Technology:Specialty Pharmacy Times Joins BPA Worldwide 25 Days to Meet ARGUS - The World's First Contactless Emotions Scanner 2Global Survey on Flow Cytometry Adoption Trends 2015 2Top Technologies in the Health and Wellness Industry 2015 2
... Market Expenditures for MRSA ... $80 Million by 2012, SUNNYVALE, Calif., Jan. 29 ... issued a medical device,license for the Xpert(TM) MRSA test ... on nasal swabs and the GeneXpert(R),System. Xpert MRSA test ...
... OXFORD, England, January 29 Circassia Ltd, ... system responses,announced today that it has successfully ... second round funding. The investment,syndicate includes new ... as existing investors, including Imperial Innovations and ...
... NanoJapan, a unique, Rice University-based program that combines ... a targeted undergraduate research internship in nanotechnology, has ... prestigious Andrew Heiskell Award for Innovation in International ... to promote and honor the most outstanding initiatives ...
Cached Biology Technology:Cepheid Receives Health Canada Medical Device License for Xpert MRSA(TM) Test and GeneXpert(R) System 2Cepheid Receives Health Canada Medical Device License for Xpert MRSA(TM) Test and GeneXpert(R) System 3Cepheid Receives Health Canada Medical Device License for Xpert MRSA(TM) Test and GeneXpert(R) System 4Circassia Raises GBP11 Million ($21.8 Million) in Oversubscribed Second Round Funding 2Rice wins innovation award for international program 2
Mouse monoclonal antibody raised against a partial recombinant SCARB2. NCBI Entrez Gene ID = SCARB2...
Mouse monoclonal antibody raised against a partial recombinant FAAH. NCBI Entrez Gene ID = FAAH...
Alpha-2-antiplasmin precursor (Alpha-2-plasmin inhibitor) (Alpha-2-PI) (Alpha-2-AP). [Source:Uniprot/SWISSPROT;Acc:P08697] Antigen: Recombinant Protein Epitope Signature Tag (PrEST)....
Mouse polyclonal antibody raised against a partial recombinant CSRP3. NCBI Entrez Gene ID = 8048...
Biology Products: