Gene Therapy for Hunter Syndrome
This week, the BBC reported on a successful clinical trial of a gene therapy to treat mucopolysaccharidosis type II, (MPS II or Hunter Syndrome). Hunter Syndrome is a genetic condition in which there is a IDS gene is faulty and the body is unable to produce the enzyme that to break down complex sugar molecules, resulting in learning difficulties, and problems in lungs, bone and in hearing. People with Hunter Syndrome typically have a shortened life expectancy, with severe cases ranging from 10-20 years.
Current treatments involve enzyme replacement, however this new trial involves taking stem cells from the patient, and inserting a working copy of the IDS gene (using a lentiviral vector), at which point the stem cells are returned to the patient, allowing them to grow more cells that are able to produce the functional enzyme.
The trial was built on the foundation of a preclinical trial conducted in mice by the same group in Manchester, UK and published in 2023. This study was conducted as described above, and the effects were measured over a period of 12-16 months, showing sustained efficacy. Treated mice maintained complex sugar clearance in the brain and other tissues, and reduced atrophy of the retinas. The treatment was found to prevent the MPSII phenotype in mice, with no post-treatment toxicity.
Please read, rate and review, this preclinical study below:
This trial represents not only a massive step forward in the treatment of Hunter Syndrome, but also for other genetic conditions for which this technology could be used to treat in the future.