Navigation Links
Can gene therapy cure fatal diseases in children?
Date:9/5/2012

In order for the body to function, a balance is necessary between the cells that build up the bones in our skeletons and the cells that break them down. In the disease malignant infantile osteopetrosis, MIOP, the cells that break down the bone tissue do not function as they should, resulting in the skeleton not having sufficient cavities for bone-marrow and nerves.

"Optic and auditory nerves are compressed, causing blindness and deafness in these children. Finally the bone marrow ceases to function and, without treatment, the child dies of anaemia and infections", explains Carmen Flores Bjurstrm. She has just completed a thesis which presents some of the research at the division for Molecular Medicine and Gene Therapy in Lund.

The researchers' work focuses on finding alternatives to the only treatment currently available against MIOP, namely a bone-marrow transplant. This treatment can be effective, but it is both risky and dependent on finding a suitable donor.

Gene therapy requires no donor, as stem cells are taken from the patients themselves. Once the cells' non-functioning gene has been replaced with a healthy copy of itself, the stem cells are put back into the patient.

Great hopes have been placed on gene therapy as a treatment method but the work has proven to be more difficult than expected. The method is used today for certain immunodeficiency diseases, and has also been applied to a blood disorder called thalassemia.

"So far, the method is not risk-free. Since it is impossible to control where the introduced gene ends up, there is a certain risk of it ending up in the wrong place and giving rise to leukaemia. This is why gene therapy is only used for serious diseases for which there is no good treatment", says Carmen Flores Bjurstrm.

The Lund researchers have conducted experiments with gene therapy in both patient cells and laboratory animals. The next step is to conduct trials on patients. The trials will probably take pla
'/>"/>

Contact: Carmen Flores Bjurstrm
carmen.flores@med.lu.se
0046-462-220-590
Lund University
Source:Eurekalert

Page: 1 2

Related medicine news :

1. KRAS gene mutation and amplification status affects sensitivity to antifolate therapy
2. Antibody therapy prevents gastrointestinal damage following radiation exposure in mice
3. Chemotherapy proves life-saving for some leukemia patients who fail induction therapy
4. Immunotherapy for elderly cancer patients finds new promise in drug combination
5. Therapy exploits addiction of leukemia cells
6. Intensity Modulated Radiation Therapy optimal for localized prostate cancer
7. Adding drug to standard chemotherapy provides no survival benefit for older lung cancer patients
8. Study Casts Doubt on Value of Pricey Prostate Cancer Therapy
9. 19th century therapy for Parkinsons disease may help patients today
10. New Psoriasis Drugs Not Much Better Than Standard Therapy, Study Finds
11. Xenotransplantation as a therapy for type 1 diabetes
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/24/2014)... a famous bamboo flooring company offering bamboo flooring products ... its multiple layer bamboo panel board collection , and ... Bamboo panel is the product made from 100 percent ... features high quality selective bamboo and eco-friendly adhesives that ... consists of multiple layers of bamboo. , According to ...
(Date:12/24/2014)... -- The holidays can be a challenge for people who ... and drinks. "This is the season in which many ... Dr. David Dodick, chair of the American Migraine Foundation, said ... to think through food and beverage choices, to help reduce ... professor of neurology at the Mayo Clinic in Scottsdale, Ariz. ...
(Date:12/24/2014)... News) -- A new, injectable weight-loss drug has been ... The agency on Tuesday approved Saxenda (liraglutide) for adults ... and have at least one weight-related health condition, such ... cholesterol. Patients taking the drug, made by Novo ... regularly, the FDA noted. "Obesity is a public ...
(Date:12/24/2014)... WEDNESDAY, Dec. 24, 2014 (HealthDay News) -- Researchers say ... cells that develop into eggs and sperm. While ... this is the first time that these types of ... produced efficiently using human stem cells, according to the ... "The creation of primordial germ cells is one of ...
(Date:12/24/2014)... -- A lab technician with the U.S. Centers for ... the Ebola virus in an agency laboratory in Atlanta ... other lab workers are being checked for possible exposure, ... exposure occurred Monday when CDC scientists doing research on ... virus to another CDC lab in the same building, ...
Breaking Medicine News(10 mins):Health News:Bamboo Flooring Company Bambooindustry.com Introduces Its Multiple Layer Bamboo Panel Board Collection 2Health News:Expert Offers Tips for Preventing Holiday Migraines 2Health News:FDA Approves New Weight-Loss Drug 2Health News:FDA Approves New Weight-Loss Drug 3Health News:CDC Monitors Lab Worker for Possible Ebola Exposure 2
... MONDAY, Dec. 5 (HealthDay News) -- There,s been a large increase ... States since 2002, a trend that may help ease concerns about ... analyzed national data and found that the number of RNs aged ... fell to fewer than 110,000 by 1991 and remained low for ...
... University of Kentucky researchers recently received a $225,000 grant ... The grant gives the team $225,000 for the ... that could total $675,000. It is funded by the ... Rural Cancer Prevention Center. Published studies have shown ...
... By Serena Gordon HealthDay Reporter , MONDAY, Dec. ... with asthma is currently using a preventive medication, according to ... percent of kids during the late 1980s. Preventive ... up, and guidelines from the U.S. National Asthma Education and ...
... -- Teenagers with autism who also have epilepsy often ... The combination means that certain behaviors common among autistic ... of their faces -- could increase their risk for ... 14 percent of children with epilepsy, the rate jumps ...
... and non-obese children found that low vitamin D levels are ... risk factors for type 2 diabetes. This study was accepted ... Endocrinology & Metabolism (JCEM). High rates of vitamin ... studies have linked low vitamin D levels to cardiovascular disease ...
... , SATURDAY, Dec. 3 (HealthDay News) -- Seizures ... during menstruation or ovulation, researchers have found. ... at the University of California, Irvine, examined the trend ... among a group of women aged 19 to 50. ...
Cached Medicine News:Health News:Good News for Those Worried About U.S. Nursing Shortage 2Health News:University of Kentucky researchers awarded CDC grant to study cancer survival in Appalachia 2Health News:More Children Using Preventive Asthma Drugs: Study 2Health News:More Children Using Preventive Asthma Drugs: Study 3Health News:Low vitamin D levels may contribute to development of Type 2 diabetes 2Health News:Epileptic Seizures May Worsen During Menstrual Cycle 2
(Date:12/24/2014)... SANTA BARBARA, Calif. , Dec. 23, 2014 ... the Junior Doctors, Association of Sierra Leone ... Sierra Leone for the treatment of local ... While a new dedicated Ebola care center was ... Ebola virus, the facility is not available for local Sierra ...
(Date:12/22/2014)... , Dec. 22, 2014  CytoSorbents Corporation (OTCQB:CTSOD), ... CytoSorb® blood purification technology to help fight deadly ... 28 countries worldwide, today announced the appointment of ... FACS, as its Senior Vice President of Clinical ... Dr. Di Russo is an accomplished ...
(Date:12/22/2014)... and Markets ( http://www.researchandmarkets.com/research/h3xm6q/us ) has announced ... Market" report to their offering. ... focuses on the developments in the self-monitoring blood glucose ... Reimbursement analysis and the effects of competitive bidding have ... SMBG meters have been performed, based on the various ...
Breaking Medicine Technology:First Ebola Treatment Unit for Local Health Workers Arrives in Sierra Leone 2CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 2CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 3CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 4CytoSorbents Appoints Dr. Gregory B. Di Russo as Senior Vice President of Clinical Development 5US Self-monitoring Blood Glucose Market 2
... Sheets: SIMH)  In a shrewd execution of its business ... with Keith Houlihan, Co-founder and President of Sanomedics ... signing of a letter of intent to acquire a ... platform in sleep apnea treatment services.  The targeted acquisition ...
... Dec. 12, 2011  Inovio Pharmaceuticals, Inc. (NYSE Amex: ... and infectious diseases, announced today that Dr. J. Joseph Kim, ... at the Oppenheimer 22nd Annual Healthcare Conference taking place December ... Inovio Presentation December 13 10:50 am ET ...
Cached Medicine Technology:Sanomedics Signs Letter of Intent to Acquire Large Sleep Disorder Service Based Operation 2Inovio Pharmaceuticals to Present at Oppenheimer Investor Conference 2Inovio Pharmaceuticals to Present at Oppenheimer Investor Conference 3
... Cutter has application in multiple open or ... resection, and/or creation of anastomoses and can ... buttressing materials such as bovine pericardium., ... a total of eight firings. Different cartridges ...
... in Open Procedures with Compact Endoscopic ... Compact-Flex45 No-Knife Articulating Linear Staplers is ... line for open surgical procedures. It ... blue cartridge, a thick-tissue green cartridge, ...
... ENDO CLIP MultApplier reusable clip applier is designed ... loading unit, each of which contains eight (8) ... shaft of the instrument is approximately 12.8" (32.5 ... and use through an appropriately sized Auto Suture ...
ENDO CLIP II ML, 10 mm Pistol Grip Single Use Clip Applier with Medium - Large Super Interlock Titanium Clips...
Medicine Products: