25 Jun 20
GENE THERAPY SUCCESS STORIES
Increasingly gene therapies are giving hope to those with genetic illness and being used to treat a wide variety of conditions, that until recently, were completely without treatment.
In 2018, a gene therapy delivered by Great Ormond Street Hospital successfully treated children with the previously fatal severe combined immunodeficiency (SCID).
In 2019, Oxford Eye Hospital have used a gene therapy to cure one of the most common forms of blindness, causing age-related macular degeneration (AMD).
Developing a successful gene therapy which can be trialled on human patients takes a huge amount of research and funding. We already have a world class research team who have started the research to create a gene therapy for Cornelia de Lange Syndrome, now all we need is the funding to keep it going and perhaps 2021 will be the year we create a gene therapy for CdLS.