Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

New findings on human genome may be useful for developing specific AIDS drugs

The AIDS virus inserts its genetic material into the genome of the infected cell. Scientists of the German Cancer Research Center have now shown for the first time that the virus almost entirely spares particular sites in the human genetic material in this process. This finding may be useful for developing new, specific AIDS drugs.

13 Nov 2009

Gene therapy increases muscle size and strength with no adverse effects in monkeys: MDA

Four macaque monkeys that received injections of genes for a protein called follistatin into upper leg muscles experienced pronounced and durable increases in muscle size and strength and no adverse effects, the Muscular Dystrophy Association (MDA) announced today. The findings could have implications particularly for injured and aging people worldwide; and for tens of millions experiencing muscle loss associated with cancer, AIDs and muscle diseases.

12 Nov 2009

Gene delivery strategy to help improve muscle mass in patients with degenerative muscle disorders

Severe weakness of the quadriceps is a defining feature of several neuromuscular disorders. Researchers at Nationwide Children's Hospital have shown that a gene delivery strategy that produces follistatin - a naturally occurring protein that inhibits myostatin, a growth factor expressed specifically in skeletal muscle - directly to the quadriceps of non-human primates results in long-term gene expression with muscle enhancing effects, including larger muscles with greater strength.

12 Nov 2009

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that it has successfully treated Duchenne muscular dystrophy (DMD) in an animal model with its proprietary gene therapy. The proof of concept studies were performed in collaboration with the group of Professor Irene Bozzoni (University of Rome, La Sapienza, Italy) and demonstrated effectiveness in the heart as well as in skeletal muscles.

11 Nov 2009

Minimally invasive surgical techniques as effective as traditional open surgery in treating rectal cancers

Laparoscopic surgery has been used in the treatment of intestinal disorders for close to 20 years, but its benefits have only recently begun to be extended to people with rectal cancer. In a prospective study of 103 patients who underwent straightforward or "hand-assisted" laparoscopic surgery for rectal cancer, a team of colon and rectal surgeons at NewYork-Presbyterian Hospital/Weill Cornell Medical Center has shown that the minimally invasive approach can be as effective as traditional open surgery in treating rectal cancers.

11 Nov 2009

Study suggests a new improved treatment option for children with Hurler's syndrome

Researchers have transplanted genetically modified hematopoietic stem cells into mice so that their developing red blood cells produce a critical lysosomal enzyme - preventing or reducing organ and central nervous system damage from the often-fatal genetic disorder Hurler's syndrome.

10 Nov 2009

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Genetix Pharmaceuticals, a leader in gene therapy of somatic stem cells, announced today the company’s clinical program to treat Adrenoleukodystrophy (ALD). The company is developing a gene therapy product in collaboration with Professor Patrick Aubourg and Doctor Nathalie Cartier of the National Institute of Health and Medical Research (INSERM).

7 Nov 2009

Gene therapy saves two children with adrenoleukodystrophy

The ELA association and Zinedine Zidane, its emblematic ambassador, are proud to announce a world premiere: the results regaring the gene therapy in adrenoleukodystrophy conducted in France have just been published in the prestigious journal Science. Two children have been treated and their diseases have been halted. The children are doing well, which is unexpected for a disease destroying the brain in a few months. This discovery opens up treatment perspectives for numerous widespread diseases.

6 Nov 2009

Boosting NAD+ after spinal cord injury may prevent permanent nerve death

Substances naturally produced by the human body may one day help prevent paralysis following a spinal cord injury, according to researchers at Weill Cornell Medical College. A recent $2.5 million grant from the New York State Spinal Cord Injury Research Board will fund their research investigating this possibility.

6 Nov 2009

New biosensor device may help in combating cancer

A powerful new biosensor developed by European researchers will help identify cells in the immune system that actively suppress tumour growth, then put them to use. Enlisting the patient's own immune system would be like sending reinforcements for resistance fighters.

5 Nov 2009

Therapy with EBV-specific CTLs effective for severely immune-compromised patients

Specially developed immune system cells that target the common Epstein-Barr virus can protect immune-suppressed bone marrow transplant recipients against lymph system disease and cancers that arise from the viral infection, said a group of researchers led by those from Baylor College of Medicine, The Methodist Hospital and Texas Children's Hospital.

3 Nov 2009

Immunotherapy, a new therapeutic area to destroy tumor cells

Targeted immunotherapy has been an attractive new therapeutic area for a number of cancers because it has the potential to destroy tumor cells without damaging surrounding normal tissue. New study results demonstrate high success rates using specialized white blood cells to prevent or treat lymphoma associated with the Epstein-Barr virus (EBV-lymphoma) in patients who have received a hematopoietic stem cell transplant (HSCT).

31 Oct 2009

Study: Gene therapy can reduce symptoms of Huntington's disease

Researchers at the California Institute of Technology (Caltech) have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models.

31 Oct 2009

USC to develop stem cell-based treatment for age-related macular degeneration with CIRM grant

Physician-researchers at the University of Southern California (USC) received a nearly $16 million grant from the California Institute of Regenerative Medicine (CIRM) to fund the development of a stem cell-based treatment for age-related macular degeneration, the leading cause of vision loss and blindness among the elderly.

29 Oct 2009

Sangamo BioSciences awarded a grant to develop zinc finger nuclease-based stem cell therapy for AIDS

Sangamo BioSciences, Inc. announced today that the California Institute for Regenerative Medicine (CIRM) has granted a $14.5 million Disease Team Research Award to develop an AIDS-related lymphoma therapy based on the application of its zinc finger nuclease (ZFN) gene-editing technology in stem cells.

29 Oct 2009

USC researchers receive grant to develop stem cell-based treatment for age-related macular degeneration

29 Oct 2009

AMT inks agreement with Progenika Biopharma for development of LPLchip

Amsterdam Molecular Therapeutics (AMT) and Progenika Biopharma announced today that the companies have entered into a development and commercialization agreement for LPLchip(TM), a diagnostic tool to rapidly diagnose patients with complete and partial lipoprotein lipase deficiency (LPLD). AMT has developed Glybera(R) as a gene therapy for patients with this disease.

29 Oct 2009

New anti-cancer medications may help in managing cancerous conditions

Bold new strategies in the battle against cancer may turn forms of the disease that presently are incurable into manageable conditions that can be controlled for long periods of time, according to an article in the current issue of Chemical & Engineering News, ACS' weekly newsmagazine.

29 Oct 2009

Moores UCSD Cancer Center awarded $20 million for cancer research

Researchers led by Moores UCSD Cancer Center Director Dennis A. Carson, MD, professor of medicine, and Catriona Jamieson, MD, PhD, assistant professor of medicine and director of the Cancer Stem Cell Research Program at the Moores UCSD Cancer Center have been awarded $20 million over four years to develop novel drugs against leukemia stem cells.

28 Oct 2009

Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Results from a breakthrough study, conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind.

27 Oct 2009

Gene Therapy News and Research

Further Reading

New findings on human genome may be useful for developing specific AIDS drugs

Gene therapy increases muscle size and strength with no adverse effects in monkeys: MDA

Gene delivery strategy to help improve muscle mass in patients with degenerative muscle disorders

AMT's gene therapy successful in treating Duchenne muscular dystrophy

Minimally invasive surgical techniques as effective as traditional open surgery in treating rectal cancers

Study suggests a new improved treatment option for children with Hurler's syndrome

Genetix Pharmaceuticals develops a gene therapy product to treat ALD

Gene therapy saves two children with adrenoleukodystrophy

Boosting NAD+ after spinal cord injury may prevent permanent nerve death

New biosensor device may help in combating cancer

Therapy with EBV-specific CTLs effective for severely immune-compromised patients

Immunotherapy, a new therapeutic area to destroy tumor cells

Study: Gene therapy can reduce symptoms of Huntington's disease

USC to develop stem cell-based treatment for age-related macular degeneration with CIRM grant

Sangamo BioSciences awarded a grant to develop zinc finger nuclease-based stem cell therapy for AIDS

USC researchers receive grant to develop stem cell-based treatment for age-related macular degeneration

AMT inks agreement with Progenika Biopharma for development of LPLchip

New anti-cancer medications may help in managing cancerous conditions

Moores UCSD Cancer Center awarded $20 million for cancer research

Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Industry focus eBook - Cell and gene therapy (2nd edition) eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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