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:9/18/2014)... is stored in DNA, tiny strands of nucleic acid ... To express this genetic data, our DNA is copied ... proteins that perform tasks in our cells. , ... RNA molecule. Unlike all other known RNAs, this molecule ... molecules are abundant, little has been known about how ...
(Date:9/18/2014)... 18, 2014Many Americans across racial and ethnic groups ... impact on their day-to-day life, more so than ... and speech (57% of African-Americans, 49% of non-Hispanic ... When asked which disease or ailment is the ... first among African-Americans followed by AIDS/HIV. Hispanics ...
(Date:9/18/2014)... The tropical rabbitfish which have devastated algal forests in ... the entire Mediterranean basin if their distribution continues to ... The study, by an international team of researchers led ... Fiona Tomas of the Mediterranean Institute for Advanced Studies ... Ecology ., Members of the team surveyed more than ...
Breaking Biology News(10 mins):Scientists find how mysterious 'circular RNA' is formed, claim muscular dystrophy link 2Americans rate losing eyesight as having greatest impact on their lives 2Americans rate losing eyesight as having greatest impact on their lives 3Tropical fish a threat to Mediterranean Sea ecosystems 2
... tobacco budworm moth and its close cousin, you wouldn,t be ... males of each species as far as six car-lengths away ... they easily find their way to mates from their own ... genes that keep the species sexually isolated are no longer ...
... to a University of Illinois study, omega-3 fatty acids may ... may have implications for treating male infertility. "In our ... for an enzyme important in making docosahexaenoic acid (DHA). In ... few if any misshaped sperm that can,t get where they ...
... do birds divorce? What makes them cheat on their spouses? ... book by a York University professor delves into these and ... The Bird Detective, Bridget Stutchbury roams forests and jungles studying ... the science behind their surprisingly sophisticated and often amusing behavior. ...
Cached Biology News:Gene studies lead to kissing cousins 2Gene studies lead to kissing cousins 3U of I study: Lack of omega-3 fatty acid linked to male infertility 2Sex, adultery, betrayal, divorce -- right in your own backyard 2
(Date:9/19/2014)... , September 19, 2014 Biotech ... development of emerging drug systems and advanced Laboratory Instrumentation. ... and Company (NYSE: BDX ), Thermo Fisher ... Inc. (NASDAQ: RGDO ), Sangamo Biosciences Inc. ... HOLX) Pressure BioSciences, Inc. (OTCQB: PBIO) ("PBI" ...
(Date:9/19/2014)... St. Paul, Minnesota (PRWEB) September 19, 2014 ... representatives from related food industries will gather October ... Meeting at the Rhode Island Convention Center in ... exhibition will feature the work of food scientists, ... development, quality control, and food production management. To ...
(Date:9/18/2014)... HealthTronics, Inc ., a leading ... and services, announced that a poster related to ... external beam radiotherapy (EBRT) is being presented at ... (MAAUA) Annual Meeting in Baltimore, Maryland September 18-21, ... and External Beam Radiation as Treatments for Localized ...
(Date:9/18/2014)... Ireland , September 18, 2014 ... and Markets ( http://www.researchandmarkets.com/research/czvg3s/biocompatible ) has ... "Biocompatible Materials - Global Strategic Business ... ,     (Logo: http://photos.prnewswire.com/prnh/20130307/600769) ,This report analyzes ... in US$ Million by the following ...
Breaking Biology Technology:Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 2Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 3Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 4Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 5Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 6Medical Instrumentation Automation Advances for Biological Sample Preparation - Company Expects Q4 Revenue Impact from Newest Instrument Systems 7Latest Research on Cereal Grain Quality, Health Benefits and Food Production to be Presented During the AACC International Annual Meeting 2Latest Research on Cereal Grain Quality, Health Benefits and Food Production to be Presented During the AACC International Annual Meeting 3HealthTronics Announces Data Comparing Cryotherapy Versus Radiation for Treating Prostate Cancer Poster Presented at the American Urological Association Regional Meeting 2Biocompatible Materials - Global Strategic Business Report 2014 2Biocompatible Materials - Global Strategic Business Report 2014 3
... in Europe, NEWPORT NEWS, Va., May 6 ... (Conformite Europeenne) approval for the,commercial sale of the Dilon ... system is a high-resolution gamma camera that enables,molecular imaging ... cancer,detection., "I am delighted that our high technology ...
... Medical Corporation,(OTC Bulletin Board: CONX), a worldwide developer and ... on Thursday, May 15, 2008,at 4:00 PM EDT to ... quarter 2008 and the status of the Company,s product,development ... in hearing management,s,discussion of the quarterly results to join ...
... BERKELEY HEIGHTS, N.J., May 6 Genta,Incorporated (Nasdaq: ... has received,notification from the NASDAQ Listing Qualifications Panel ... to sustain compliance with the,minimum stockholders, equity requirement ... Panel has also determined that de-listing from,The NASDAQ ...
Cached Biology Technology:Genta Announces Pending Transfer of its Common Stock to the Over-the-Counter Bulletin Board 2Genta Announces Pending Transfer of its Common Stock to the Over-the-Counter Bulletin Board 3
Human peripheral blood eosinophils, For immunohistochemistry (IHC) Cell Chip with Human Peripheral Blood Granulocytes...
Human peripheral blood PBMCs, For immunohistochemistry (IHC) Cell Chip with Human Peripheral Blood PBMC and T Cells...
Cord blood endothelial progenitor cells, For immunohistochemistry (IHC) Cell Chip with Human Umbilical Cord Blood Progenitor Cells...
Human plasmacytoid dendritic cells, BDCA-4+, For immunohistochemistry (IHC) Cell Chip with Human Peripheral Blood Dendritic Cells and EPCs...
Biology Products: