Category Archives: Gene therapy

ScienceDaily (Sep. 5, 2012) That low bone density causes osteoporosis and a risk of fracture is common knowledge. But an excessively high bone density is also harmful. The most serious form of excessively high bone density is a rare, hereditary disease which can lead to the patient's death by the age of only five. Researchers at Lund University in Sweden are now trying to develop gene therapy against this disease.

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 anemia 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 leukemia. 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 place at the hospital in Ulm, Germany, which currently treats the majority of children in Europe suffering from MIOP.

MIOP is a rare disease: in Sweden a child is born with the condition approximately once every three years. Worldwide, the incidence of the disease is one case for every 300 000 births. It is, however, more common in Costa Rica where 3-4 children per 100 000 births have the disease.

"But there are several other genetic mutations that lead to other osteopetrosis diseases. If we manage to treat MIOP, it may become possible to treat these other conditions as well," hopes Carmen Flores Bjurstrm along with her supervisor, Professor Johan Richter.

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Can gene therapy cure fatal diseases in children?

Take a close look at the athletes competing in this year's Summer Olympic Games in London --their musculature will tell you a lot about how they achieved their elite status. Endless hours of training and commitment to their sport played a big role in building the bodies that got them to the world's premier athletic competition. Take an even closer look--this one requires microscopy--and you'll see something else, something embedded in the genetic blueprints of these young men and women that's just as important to their success. [More]

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http://rss.sciam.com/sciam/topic/gene-therapy

From Nature magazine

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http://rss.sciam.com/sciam/topic/gene-therapy

One day in early 1995 a man named bob massie walked into my office at the outpatient clinic of Massachusetts General Hospital in Boston. Massie told me he had been infected with HIV--the virus that causes AIDS--for 16 years and yet had never shown any symptoms. My physical examination confirmed he was healthy, in stark contrast to all other patients I saw that day. At that time, a new combination of drugs was being tested that would eventually slow the progressive decline in immune function that HIV caused. In 1995, however, most people who had been infected with HIV for a decade or more had already progressed to AIDS--the stage marked by the inability to fight off other pathogens. The young man standing before me had never taken anti-HIV medication and strongly believed that if I learned the secret to his good fortune, the information could help others to survive what was then generally thought to be a uniformly fatal disease.

[More]

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http://rss.sciam.com/sciam/topic/gene-therapy

Tears and a runny nose can be unpleasant on a windy day, but these mucosal secretions play a vital role in protecting the body from viruses and other malicious microbes. Unfortunately, mucus is also adept at washing away medication designed to treat infections and inflammation that occur when an infectious agent is successful in penetrating the body's defenses [More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

Take a close look at the athletes competing in this year's Summer Olympic Games in London --their musculature will tell you a lot about how they achieved their elite status. Endless hours of training and commitment to their sport played a big role in building the bodies that got them to the world's premier athletic competition. Take an even closer look--this one requires microscopy--and you'll see something else, something embedded in the genetic blueprints of these young men and women that's just as important to their success. [More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

From Nature magazine

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

One day in early 1995 a man named bob massie walked into my office at the outpatient clinic of Massachusetts General Hospital in Boston. Massie told me he had been infected with HIV--the virus that causes AIDS--for 16 years and yet had never shown any symptoms. My physical examination confirmed he was healthy, in stark contrast to all other patients I saw that day. At that time, a new combination of drugs was being tested that would eventually slow the progressive decline in immune function that HIV caused. In 1995, however, most people who had been infected with HIV for a decade or more had already progressed to AIDS--the stage marked by the inability to fight off other pathogens. The young man standing before me had never taken anti-HIV medication and strongly believed that if I learned the secret to his good fortune, the information could help others to survive what was then generally thought to be a uniformly fatal disease.

[More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

Tears and a runny nose can be unpleasant on a windy day, but these mucosal secretions play a vital role in protecting the body from viruses and other malicious microbes. Unfortunately, mucus is also adept at washing away medication designed to treat infections and inflammation that occur when an infectious agent is successful in penetrating the body's defenses [More]

Source:
http://rss.sciam.com/sciam/topic/gene-therapy

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/kxltqj/gene_therapy_marke) has announced the addition of the "Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory Surveillance and High Compliance Standards" report to their offering.

Gene Therapy: the Next Big Step in Cancer Treatments.

The fight against cancer is leading a new movement in gene therapy, as the failure of conventional cancer therapies is fuelling demand for new treatments, according to a new report by healthcare experts GBI Research.

The new report* states that gene therapy technology is still in its nascent stage, and high levels of regulatory surveillance in clinical development is affecting progress. However, the increasing potential of upcoming treatments and shortcomings in traditional therapies is gradually leading to broader acceptance of gene therapy in medicine.

Therapies such as chemotherapy and hormone therapy control the progression of diseases, but are often associated with severe side effects, such as nausea, hair loss and abnormal blood cell counts. Once administered, the drugs induce systemic action throughout the body, and patients often die due to the side effects of treatment rather than the cancer itself. The inability of these conventional therapies to cure diseases has created a significant unmet need in the treatment of cancer, as well as Human Immunodeficiency Virus (HIV), autoimmune diseases, and viral infections.

Targeted therapies such as monoclonal antibodies, stem cell therapies, Ribonucliec Acid (RNA) therapies and gene therapies have initially shown better efficacy and safety profiles compared to chemotherapies.

Gene therapy has several promising drug candidates, which are likely to drive the growth of the gene therapy market if clinical trials are successful. Collategene by AnGes MG, Cardium Therapeutics' Generx, and Vical Incorporation's Allovectin-7 are in development for a wide range of cancer indications, and are expected to compete in the oncology therapeutics market as the market acceptance of gene therapy improves over time.

Companies Mentioned

- ReGenX Biosciences

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Research and Markets: Gene Therapy Market to 2018 - Product Development Slowed by Clinical Failures, Close Regulatory ...