Navigation Links
Clean correction of a patient's genetic mutation
Date:10/12/2011

Using 'molecular scissors' to snip the genome at precisely the right place, they then inserted a correct version of the gene using a DNA transporter called piggyBac. The piggyBac sequences were subsequently removed from the cells, allowing them to be converted into liver cells without any trace of residual DNA damage at the site of correction.

The scientists then proved that the accurate copy of the gene was now active in the liver cells they had produced by demonstrating the presence of normal alpha1-antitrypsin protein in both test tube and mouse experiments.

"We have developed new systems to target genes and integrated all the components to correct, efficiently, defects in patient cells," says Professor Allan Bradley, Director Emeritus of the Wellcome Trust Sanger Institute. "Our systems leave behind no trace of the genetic manipulation, save for the gene correction.

"These are early steps but, if this technology can be taken into treatment, it will offer great possible benefits for patients."

"This study represents a first step toward personalised cell therapy for genetic disorders of the liver," explains Dr Ludovic Vallier, Medical Research Council (MRC) senior Fellow and Principal Investigator at the University of Cambridge's MRC Centre for Stem Cell Biology and Regenerative Medicine and Department of Surgery, who studies human pluripotent stem cell biology. "We still have major challenges to overcome before any clinical applications but we have now the tools necessary to advance toward this essential objective."

In their analysis of the stem cells, the research team showed that genomes of stem cells commonly contain mutations, whose cause remains unclear. However, the researchers were able to use the latest sequencing techniques to find cells with minimum numbers of mutations whose genetic consequences they could examine. They make clear that careful screening of stem cells will be needed to contribute to safe a
'/>"/>

Contact: Don Powell
press.office@sanger.ac.uk
44-012-234-96928
Wellcome Trust Sanger Institute
Source:Eurekalert

Page: 1 2 3

Related biology technology :

1. Purifying nanorods: Big success with tiny cleanup
2. Millstone Medical Outsourcing Acquires New Facility to Accommodate Growing Business, Provide Additional Clean Room Capacity
3. SRNLs microbes useful for for environmental cleanup and oil recovery
4. TechConnect World 2009 Matches Tomorrows Technology Solutions with Todays Business Needs for Clean-Technology, Biotechnology and Nanotechnology
5. The Elmasonic S Ultrasonic Cleaner: Designed for the Scientific Laboratory
6. San Diego universities, government and industry rally around local Clean-Tech cluster
7. China Clean Energy Resumes Biodiesel Production After Temporary Halt
8. White Paper Details How DNA Sequencing Services Can Prevent Cleanroom Shutdowns at Medical Device and Pharmaceutical Manufacturers
9. Photo-catalytic, self cleaning coating for building exteriors
10. DAZ Systems, Inc. Thrives in the Clean Energy Industry
11. ASP Announces Availability of EVOTECH(TM) Endoscope Cleaner and Reprocessor - First Commercially Available System to Eliminate Manual Cleaning of Endoscopes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:7/11/2014)... -- , Not for release, publication or distribution ... jurisdiction where to do so would constitute a violation of ... ("Shire" or the "Company") (LSE: SHP, NASDAQ: SHPG ... has held a meeting with representatives of AbbVie. ... agreement or approval of AbbVie. A further update ...
(Date:7/11/2014)... July 11, 2014 /CNW/ - Sunovion Pharmaceuticals Canada Inc. ... (eslicarbazepine acetate) for use as a once-daily adjunctive ... with epilepsy who are not satisfactorily controlled with conventional ... in patients under 18 years of age. ... disorders and according to Epilepsy Canada, it affects 0.6% ...
(Date:7/10/2014)... July 10, 2014 Product and ... very low abundance and are often “lost in ... time-consuming. , Join presenters Dr. Rowel Tobias, Senior ... John Anders, Head of Quality at Nanotherapeutics, Inc., ... that can speed detection and quantitation while achieving ...
(Date:7/10/2014)... CA (PRWEB) July 10, 2014 Unraveling ... closer together. The Archer Family purchased a ... within DNA, but ended up with a lifetime of ... contained within the family members’ DNA genuinely brought the ... decided to search deeper into genetic history and prior ...
Breaking Biology Technology:Shire plc - Statement re: Media Speculation 2Sunovion Pharmaceuticals Canada Inc. Announces Health Canada Approval of APTIOM(TM) (eslicarbazepine acetate) as a Once-Daily Adjunctive Treatment for Partial-Onset Seizures in Patients with Epilepsy 2Sunovion Pharmaceuticals Canada Inc. Announces Health Canada Approval of APTIOM(TM) (eslicarbazepine acetate) as a Once-Daily Adjunctive Treatment for Partial-Onset Seizures in Patients with Epilepsy 3Sunovion Pharmaceuticals Canada Inc. Announces Health Canada Approval of APTIOM(TM) (eslicarbazepine acetate) as a Once-Daily Adjunctive Treatment for Partial-Onset Seizures in Patients with Epilepsy 4Novel Ms Techniques Speed Detection of Biopharmaceutical Product and Process Impurities, New Webinar Hosted by Xtalks 2Ancestry DNA Test Reveals a Controversial and Undiscovered Family History 2Ancestry DNA Test Reveals a Controversial and Undiscovered Family History 3Ancestry DNA Test Reveals a Controversial and Undiscovered Family History 4Ancestry DNA Test Reveals a Controversial and Undiscovered Family History 5
... Therapeutic Leaders Join Forces to Promote Early Detection ... and Treatment of Hypertension in the UK, ... ,InterCure, Ltd. (Tel Aviv Stock Exchange: INCR), and Omron Healthcare ... an,agreement to strengthen the link between hypertension diagnostics and,therapies in ...
... Fisher,Scientific Inc. (NYSE: TMO ) announced today ... and chief executive,officer, are adopting stock- trading programs ... the future sale of company stock. Mr.,Dekkers, who ... August,15-17, 2007, has adopted a plan for the ...
... DIEGO, Aug. 20 Vical Incorporated,(Nasdaq: VICL ... team with,the appointment of Andrew R. de Guttadauro ... Vical, Mr. de Guttadauro was Senior Director of,Strategy ... two-year,assignment overseeing the commercial development of that company,s ...
Cached Biology Technology:InterCure and Omron Healthcare (UK) Ltd. Announce Marketing Collaboration for Hypertension Market in United Kingdom 2InterCure and Omron Healthcare (UK) Ltd. Announce Marketing Collaboration for Hypertension Market in United Kingdom 3InterCure and Omron Healthcare (UK) Ltd. Announce Marketing Collaboration for Hypertension Market in United Kingdom 4Thermo Fisher Scientific Executive Officers Adopting SEC Rule 10b5-1 Trading Programs 2Vical Names Andrew de Guttadauro Vice President, Corporate Development 2Vical Names Andrew de Guttadauro Vice President, Corporate Development 3
(Date:7/10/2014)... CORAL SPRINGS, Florida , July 1, 2014 ... smart wallets pave way for convenience and improved security: NXT-ID, ... Google Inc. (NASDAQ: GOOG ), Apple Inc. (NASDAQ: ... ) and MasterCard Corporation (NYSE: MA) NXT-ID, Inc., (OTCQB: ... market, is pleased to announce that the second series of ...
(Date:7/10/2014)... NXT-ID, Inc., (OTCQB: NXTD) a biometric authentication company focused on ... Pereira was interviewed on July 1 st on ... . Mr. Pereira discusses the company,s next generation smart ... how the Wocket™ aims to replace a traditional wallet with ... Harris tells Gino he has never heard of the ...
(Date:7/10/2014)... GOTHENBURG, Sweden , July 7, 2014 ... won its first major design win (DW). A Chinese Top ... with the planned start of mass production in October 2014. ... with a Chinese Top 5 smartphone OEM, which has a ... 2014 and a target sales volume for this phone of ...
Breaking Biology News(10 mins):Advantages for Digital Wallets Eliminate Need to Carry Credit Cards - Large Scale CNBC Ad Campaign for Latest Smart Wallet to Commence July 7th 2Advantages for Digital Wallets Eliminate Need to Carry Credit Cards - Large Scale CNBC Ad Campaign for Latest Smart Wallet to Commence July 7th 3Advantages for Digital Wallets Eliminate Need to Carry Credit Cards - Large Scale CNBC Ad Campaign for Latest Smart Wallet to Commence July 7th 4Advantages for Digital Wallets Eliminate Need to Carry Credit Cards - Large Scale CNBC Ad Campaign for Latest Smart Wallet to Commence July 7th 5Security and Your Money: Tampa Bay Radio Host Cedric Harris Talks to NXT-ID CEO Mr. Gino Pereira about the Wocket Smart Wallet 2Security and Your Money: Tampa Bay Radio Host Cedric Harris Talks to NXT-ID CEO Mr. Gino Pereira about the Wocket Smart Wallet 3Security and Your Money: Tampa Bay Radio Host Cedric Harris Talks to NXT-ID CEO Mr. Gino Pereira about the Wocket Smart Wallet 4FPC Wins DW for 1021 Touch Sensor for Flagship Smartphone from Chinese Top 5 Smartphone OEM 2
... Sept. 27, 2010 If drivers are yakking on cell phones and ... or navigation system, they still can get directions from devices that are ... fingertips left or right, a University of Utah study found. ... researchers say they don,t want their results to encourage dangerous and distracted ...
... of a molecular communications pathway that influences the ... to targeted therapies for improving bone marrow transplant ... approach, researchers at Cincinnati Children,s Hospital Medical Center ... that pharmacological inhibition of a signaling pathway triggered ...
... and Oxford, UK, 27 September 2010: Research published this ... method of enzyme-controlled movement of a single strand of ... researchers at the University of California Santa Cruz (UCSC), ... strands. The publication describes the observation of single stranded ...
Cached Biology News:Let your fingers do the driving 2Let your fingers do the driving 3Let your fingers do the driving 4Let your fingers do the driving 5Inhibiting cell signaling pathway may improve bone marrow transplant success rate 2Nature Nanotechnology paper shows enzyme-controlled movement of DNA polymer through a nanopore 2
...
Rabbit polyclonal to MCP2 ( Abpromise for all tested applications). entrezGeneID: 6355 SwissProtID: P49873...
Mouse monoclonal antibody raised against a partial recombinant AATK. NCBI Entrez Gene ID = AATK...
... AS-2057 are fully automatic sample injection systems ... of precision. Sampling flexibility is unparalleled with ... automation and combinatorial chemistry. Also available is ... standard rack for 50 2,ml vials. The ...
Biology Products: