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【medical-news】首个临床试验基因心衰治疗
NewYork-Presbyterian Hospital/Columbia University Medical Center is the only center in the New York City area where the therapy is currently available.
Patients enrolled in the multicenter CUPID trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) will undergo a minimally invasive cardiac catheterization procedure that will introduce a specially engineered gene that stimulates production of an enzyme necessary for the heart to pump more efficiently.
"This new therapy seeks to replenish the levels of this enzyme by introducing the gene for SERCA2a, which is depressed in these patients. If proven effective, this approach could be an alternative to heart transplant for patients without any other options," says Dr. Donna Mancini, the study's principal investigator at NewYork-Presbyterian Hospital/Columbia University Medical Center, where she is medical director of cardiac transplantation. She also is professor of medicine at Columbia University College of Physicians and Surgeons.
Gene therapy is a technique for correcting defective genes responsible for disease development by inserting genes into a patient's cells and tissues. In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal" disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a non-pathogenic virus most people have been exposed to in adolescence that has been genetically altered to carry normal human DNA.
More than five million people in the U.S. have heart failure. Patients with severe form of the disease have trouble breathing because the heart cannot pump fluid out of their lungs. Seventy percent die of the disease within 10 years, and the five-year survival rate is less than 50 percent. Heart failure is the only cardiovascular disease whose incidence has been increasing rather than decreasing.
The multicenter national trial is funded and administered by the Celladon Corporation of La Jolla, Calif. The company has reported that the therapy, called MYDICAR®, has been shown to lead to significant improvements in heart function without significant safety concerns in large-animal models of heart failure. This is the first human trial of this technique.
来源：http://www.sciencedaily.com/releases/2008/06/080618145938.htm 没人认领吗 本人已认领该文编译，48小时后若未提交译文，请其他战友自由认领。 Could injecting a gene into a patient with severe heart failure reverse their disabling and life-threatening condition? Physician-scientists are setting out to answer that question in a first-ever clinical trial of gene therapy to treat severe heart failure.
将某个基因注射进严重心衰的患者身体，能逆转他们糟糕的生活质量和生命经常受到威胁的现状吗？医学科学家正着手通过基因疗法治疗严重心衰患者的临床试验来回答这个问题。
NewYork-Presbyterian Hospital/Columbia University Medical Center is the only center in the New York City area where the therapy is currently available.
纽约-Presbyterian 医院/哥伦比亚大学医学中心是目前纽约地区唯一一个可以进行这种治疗的地方。
Patients enrolled in the multicenter CUPID trial (Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease) will undergo a minimally invasive cardiac catheterization procedure that will introduce a specially engineered gene that stimulates production of an enzyme necessary for the heart to pump more efficiently.
征集到多中心CUPID（心血管疾病的经皮静脉钙上调基因疗法）临床试验的患者，需要接受一项侵袭性很小的心导管安置手术，通过它可以导入特别设置的目的基因，这种基因能刺激一种能让心脏跳的更有效率的酶产量。
"This new therapy seeks to replenish the levels of this enzyme by introducing the gene for SERCA2a, which is depressed in these patients. If proven effective, this approach could be an alternative to heart transplant for patients without any other options," says Dr. Donna Mancini, the study's principal investigator at NewYork-Presbyterian Hospital/Columbia University Medical Center, where she is medical director of cardiac transplantation. She also is professor of medicine at Columbia University College of Physicians and Surgeons.
“这项新疗法是通过导入SERCA2a基因，以恢复该基因特定编译产物——酶的水平，这种酶在患者体内是下降的。如果证实有效，这种方法将被用来作为别无选择的心脏移植的替代疗法，”纽约-Presbyterian医院/哥伦比亚大学医学中心心脏移植主任，本研究的主要观察员，Donna Mancini博士如是说。她同时也是哥伦比亚大学内外科教授。
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作者:admin@医学,生命科学 2011-08-12 17:19
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