Gene Therapy for Leber's Congenital Amaurosis (LCA)

Filed in archive Gene Therapy on October 26, 2009

Usign a single shot of gene therapy, scientists were able to improve the vision in five children and seven adults with Leber's congenital amaurosis (LCA), an inherited retinal degenerative disease characterized by severe loss of vision at birth. Although the patients did not attain normal eyesight, half of them (six of 12) improved enough that they may no longer be classified as legally blind.

The 12 subjects ranged in age from 8 to 44 years old at the time of treatment. Four of the children, aged 8 to 11, are the world’s youngest individuals to receive gene therapy for a non-lethal disease (A fifth subject was 17 years old). On the other end of the age scale, the 35-year-old man and 44-year-old woman are the oldest patients to ever receive gene therapy for retinal degeneration.
For the current human trial, the research team used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene, called RPE65, that is mutated in one form of LCA, called LCA2, that accounts for 8 to 16 percent of all LCA cases. Jeannette Bennicelli, Ph.D., in Bennett’s laboratory, cloned the gene. The clinical vector production facility at Children’s Hospital’s Center for Cellular and Molecular Therapeutics (CCMT), directed by Fraser Wright, Ph.D., manufactured the vector.

Here's a video showing the results of an obstacle course test with one patient, 3 months after injection of the left eye.



Source