Many of the gene therapy research studies were halted few years back due to the death of Jesse Gelsinger. He was the first patient to die from Gene therapy two days after the beginning of the clinical trial. But many scientists believe that gene therapy is actually a promising way to cure many rare and chronic diseases.
The treatment using Gene therapy basically means that 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 virus that has been genetically altered to carry normal human DNA.
Scientist are continuing their studies to find ways to cure disease using gene therapy. The last successful clinical trial was conducted by researchers from the University of Pennsylvania School of Medicine and the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia. They used gene therapy treatment on five children and seven adults with retinal disease called Leber’s congenital amaurosis (LCA). All of the patients showed great improvement in their sight.
One of the patients used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. But now, after a small injection that produce light-sensitive pigments in the back of his eye, he sits in front with classmates. Watch this miracle
“This study reports dramatic results in restoring vision to patients who previously had no options for treatment,” said Katherine A. High, M.D., co-first author of the study and the director of the Center for Cellular and Molecular Therapeutics, the facility that sponsored the clinical trial at The Children’s Hospital of Philadelphia. “These findings may expedite development of gene therapy for more common retinal diseases, such as age-related macular degeneration.”
Researchers in England also successfully restored eyesight for six patients with genetic diseases.
Via – ScienceDaily