Friday, July 07, 2006
Gene Therapy Using Ultrasound-Targeted Microbubble Destruction
Gene Therapy Using Ultrasound-Targeted Microbubble DestructionBy Hsien Hsien Lei, PhD Related entries in Genetic Engineering, Genetics of Disease, Genetic Ingenuity
There’s a very cool new way to deliver insulin genes to the pancreas where insulin is normally manufactured. Ultrasound-targeted microbubble destruction (UTMD) delivers the genes encapsulated within microscopic “bubbles.” When the genes have reached the pancreas, the bubbles are popped using ultrasound and the genes are released.
One of the genes that has been successful delivered using UTMD is the hexokinase I gene involved in regulating insulin. In rats, those who received UTMD gene therapy experienced an increase in insulin with a resulting decrease in blood sugar levels.
Dr. Paul Grayburn:
Not only was their blood sugar lowered, but there was no evidence of any damage to the pancreas. Other forms of gene therapy are usually invasive and unlike the UTMD technique, do not target the tissues and organs specifically.
Because the pancreas in people with type 1 diabetes is unable to produce insulin, gene therapy and regeneration of the islet cells could be the two main ways to allow patients to produce their own insulin rather than depend on externally injected insulin. I’m sure my friend Rob at Diabetes Notes thinks this technology couldn’t happen soon enough!
Medical News Today, May 23, 2006
Technorati Tags: diabetes, utmd, type 1 diabetes, ultrasound targeted microbubble destruction, gene therapy, genetics, genes, dna, disease, health
In March 2006 an international group of scientists announced the successful use of gene therapy to treat two adult patients for a disease affecting myeloid cells. The study, published in Nature Medicine, is believed to be the first to show that gene therapy can cure diseases of the myeloid system [1]
University of California, Los Angeles, research team gets genes into the brain using liposomes coated in a polymer call polyethylene glycol (PEG). The transfer of genes into the brain is a significant achievement because viral vectors are too big to get across the "blood-brain barrier." This method has potential for treating Parkinson's disease. See Undercover genes slip into the brain at NewScientist.com (March 20, 2003).
RNA interference or gene silencing may be a new way to treat Huntington's. Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced. See Gene therapy may switch off Huntington's at NewScientist.com (March 13, 2003).
New gene therapy approach repairs errors in messenger RNA derived from defective genes. Technique has potential to treat the blood disorder thalassaemia, cystic fibrosis, and some cancers. See Subtle gene therapy tackles blood disorder at NewScientist.com (October 11, 2002).
Gene therapy for treating children with X-SCID (sever combined immunodeficiency) or the "bubble boy" disease is stopped in France when the treatment causes leukemia in one of the patients. See 'Miracle' gene therapy trial halted at NewScientist.com (October 3, 2002).
Researchers at Case Western Reserve University and Copernicus Therapeutics are able to create tiny liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane. See DNA nanoballs boost gene therapy at NewScientist.com (May 12, 2002).
Sickle cell is successfully treated in mice. See Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease from March 18, 2002, issue of The Scientist.
The success of a multi-center trial for treating children with SCID held from 2000 and 2002 was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily, but resumed after regulatory review of the protocol in the United States, the United Kingdom, France, Italy, and Germany. (V. Cavazzana-Calvo, Thrasher and Mavilio 2004)
University of California, Los Angeles, research team gets genes into the brain using liposomes coated in a polymer call polyethylene glycol (PEG). The transfer of genes into the brain is a significant achievement because viral vectors are too big to get across the "blood-brain barrier." This method has potential for treating Parkinson's disease. See Undercover genes slip into the brain at NewScientist.com (March 20, 2003).
RNA interference or gene silencing may be a new way to treat Huntington's. Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced. See Gene therapy may switch off Huntington's at NewScientist.com (March 13, 2003).
New gene therapy approach repairs errors in messenger RNA derived from defective genes. Technique has potential to treat the blood disorder thalassaemia, cystic fibrosis, and some cancers. See Subtle gene therapy tackles blood disorder at NewScientist.com (October 11, 2002).
Gene therapy for treating children with X-SCID (sever combined immunodeficiency) or the "bubble boy" disease is stopped in France when the treatment causes leukemia in one of the patients. See 'Miracle' gene therapy trial halted at NewScientist.com (October 3, 2002).
Researchers at Case Western Reserve University and Copernicus Therapeutics are able to create tiny liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane. See DNA nanoballs boost gene therapy at NewScientist.com (May 12, 2002).
Sickle cell is successfully treated in mice. See Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease from March 18, 2002, issue of The Scientist.
The success of a multi-center trial for treating children with SCID held from 2000 and 2002 was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily, but resumed after regulatory review of the protocol in the United States, the United Kingdom, France, Italy, and Germany. (V. Cavazzana-Calvo, Thrasher and Mavilio 2004)
ref - wikipedia.org
