Navigation Links
Canada launches first gene therapy trial for Fabry disease
Date:1/24/2013

CALGARY Researchers in Calgary have launched the first gene therapy clinical trial in the world for Fabry disease, a rare inherited enzyme deficiency that can shorten the lifespan of people who have it by as much as 40 years.

Researchers will first remove a quantity of stem cells from a Fabry patient's blood. Then a working copy of a new gene will be inserted into the stem cells using a specially engineered virus. During the final phase of the trial, researchers hope to transplant these stem cells back into the donor patient and the new, working copy of the gene will make the missing enzyme.

The clinical trial has been prompted by promising gene therapy results in mice performed in the laboratory of Dr. Jeffrey Medin at the University Health Network in Toronto. Dr. Medin is the principal investigator of the pan-Canada team grant that is supporting this trial.

"We hope this will one day become a form of treatment that effectively cures Fabry disease," says Dr. Aneal Khan, a medical geneticist based at Alberta Children's Hospital, who is leading the Calgary segment of the national project.

"It could also help establish a platform on which we can create gene therapies for other illnesses and establish Calgary as a national leader in this experimental field of interventional genetics."

Although several gene therapies have been used in Canada for cancer, this study will be the first in the country to test a gene therapy for an inherited metabolic disorder.

People with Fabry disease have a change in a gene called GLA and can't make enough enzyme to break down a fatty substance called Gb3. The build-up of Gb3 can lead to problems in the kidneys, heart and brain. About 400 Canadians, including 25 Calgarians, have Fabry disease.

Although the project is headquartered in Toronto, physicians and scientists in Calgary will play a major role in the clinical trial. In particular, the lab at Foothills Medica
'/>"/>

Contact: Gregory Harris
gregory.harris@albertahealthservices.ca
403-619-3108
Alberta Health Services
Source:Eurekalert

Page: 1 2 3

Related biology news :

1. Disease-carrying colonizers on the move: Predicting the spread of ticks across Canada
2. Ducks Unlimited Canada and Canadian Light Source partnership to shed light on wetlands
3. Potent human toxins prevalent in Canadas freshwaters
4. Meet Xenoceratops: Canadas newest horned dinosaur
5. USAs ancient hurricane belt and the US-Canada equator
6. Inspired: Canada funds 68 bold, inventive ways to improve health, save lives in developing countries
7. LABS, Inc. Launches Suite of Next-Generation Test Offerings; Focuses on Expanding Complex Biologic Testing Portfolio in 2012
8. Elsevier launches new journal Algal Research
9. Clinical trial launches to see whether vitamin D helps treat multiple sclerosis
10. Thomson Reuters Launches Life Sciences Partner Ecosystem to Drive Collaborative R&D Drug Processes
11. WanderID Launches Breakthrough ID Product for Children, Seniors
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/15/2014)... CAMBRIDGE, Mass. (September 15, 2014) The so-called ... which makes proteinhas long provided a simplified explanation ... in living organisms. , In reality, of ... the schema first articulated nearly 60 years ago ... DNA,s double-helix structure. For one, there are multiple ...
(Date:9/15/2014)... Reproductive cell division has evolved a simple, mechanical ... in the Sept. 11 Science Express. ... chromosome counts and misalignments that lead to infertility, ... reproductive cell division cause these problems, but what ... Adele Marston of the Wellcome Trust Center for ...
(Date:9/15/2014)... of microorganisms may be responsible for much of the ... for the global carbon cycle and climate change. , ... most life on this planet, it is only produced ... is so large and complex. For humans, vitamin B12 ... nervous systems, as well as DNA synthesis in cells ...
Breaking Biology News(10 mins):Scientists discover RNA modifications in some unexpected places 2Cells simply avoid chromosome confusion 2Cells simply avoid chromosome confusion 3Researchers discover new producer of crucial vitamin 2
... key influence on a child,s ability to deal with stress. ... an important role in changing the impact of genes that ... , That,s the message from a new study by ... University, the University of North Carolina-Greensboro, and North Carolina State ...
... than 125 years ago Charles Darwin first reported that most ... day and this week, scientists are reporting the discovery ... growth spurts, and the ability of plants to move in ... circadian rhythms the "biological clock" genes that are influenced ...
... of researchers headed by biologists at Washington University in St. ... manages two disparate operations photosynthesis and nitrogen fixation ... Himadri B. Pakrasi, Ph.D., George William and Irene Koechig ... the genome of Cyanothece sp. ATCC 51142 to ...
Cached Biology News:Early parenting plays key role in infants' physiological response to stress 2'Biological clock' genes control plant growth 2Cyanobacterium sequenced features rare linear chromosome 2Cyanobacterium sequenced features rare linear chromosome 3
(Date:9/15/2014)... September 16, 2014 Cancer ... and Adoptive T-cell Therapies is a new market ... on the three principal types of therapeutics that ... development in cancer immunotherapy (which is often called ... vaccines and Adoptive cellular immunotherapy. , The ...
(Date:9/15/2014)... Researchers from North Carolina State University have developed a ... by applying very low voltages, opening the door to ... other technologies. The technique hinges on the fact that ... be deposited or removed acts as a surfactant, ... surrounding fluid. , The researchers used a liquid metal ...
(Date:9/15/2014)... Northeastern University has developed a novel method for ... of nanocarbon structures in carbon nanotube arrays. The ... allow the researchers to tailor the physical properties ... from electronic devices to CNT-reinforced composite materials found ... Their findings were published in the journal ...
(Date:9/15/2014)... -- iVinci Health LLC, the company behind the healthcare industry,s ... as VisitPay®, has successfully closed $5 million in new ... new extension to VisitPay® – an online Payment and ... for patients to manage their medical bills and set-up ... families. The new round of funding, which ...
Breaking Biology Technology:Cancer Immunotherapy Market: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies 2Cancer Immunotherapy Market: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies 3Cancer Immunotherapy Market: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies 4Cancer Immunotherapy Market: Immune Checkpoint Inhibitors, Cancer Vaccines, and Adoptive T-cell Therapies 5Researchers control surface tension to manipulate liquid metals 2Northeastern University Researchers Develop Novel Method for Working with Nanotubes 2iVinci Health Raises $5M to Fuel National Expansion, Announces Launch of Personal Finance Portal for Healthcare 2iVinci Health Raises $5M to Fuel National Expansion, Announces Launch of Personal Finance Portal for Healthcare 3
... the world,s leading peer-reviewed research journal, is partnering with Applied Biosystems, ... Advances in Cancer Genomics to be broadcast live online at 12 ... ... (PRWEB) April 15, 2009 -- The business office of Science, the ...
... Technologies, Inc., a leader in adult stem cell research and manufacturing, ... Scientific Advisory Board. In this capacity, she will help Stemedica ... cellular delivery system for burn and wound management. , ... ...
... EAST RUTHERFORD, N.J., April 14 Cambrex Corporation (NYSE: ... notified by the New York Stock Exchange (the "NYSE") that ... the Company,s total market capitalization and its stockholders, equity. ... the NYSE that it did not meet its continued listing ...
Cached Biology Technology:Science Magazine to Host Webinar on Advances in Cancer Genomics 2Science Magazine to Host Webinar on Advances in Cancer Genomics 3Stemedica Signs Scientific Advisory Board Agreement with Leading Pediatric Burn & Wound Care Researcher 2Stemedica Signs Scientific Advisory Board Agreement with Leading Pediatric Burn & Wound Care Researcher 3Stemedica Signs Scientific Advisory Board Agreement with Leading Pediatric Burn & Wound Care Researcher 4Cambrex Receives Notice Regarding NYSE Listing 2Cambrex Receives Notice Regarding NYSE Listing 3