Italian scientists found a way to harness gene therapy vectors from HIV to cure two severe and rare genetic diseases - metachromatic leukodystrophy and Wiskott-Aldrich syndrome.

San Raffaele-Telethon Institute for Gene Therapy or TIGET researchers and Telethon in Italy changed how we think on HIV. Director Luigi Naldini led the team and found something new about the deadly virus which allows them to treat six children using HIV-derived genetic messengers.

"The idea of using HIV vector came many years ago to Dr. Luigi Naldini and his team. They realised that the vector had a unique property which is highly efficient in introducing information into cell's specific DNA," Alessandra Biffi, MD and group leader at TIGET, said in a press release.

The gene therapy was used to treat three children with metachromatic leukodystrophy and three others with Wiskott-Aldrich syndrome. Three years after the treatment, all of them are now well and benefited significantly from the therapy.

"The results obtained from the first six patients are very encouraging: The therapy is not only safe, but also effective and able to change the clinical history of these severe diseases," said TIGET Director Luigi Naldini in Milan in a press release.

Metachromatic Leukodystrophy

Metachromatic Leukodystrophy or MLD is a metabolic disorder involving lysosomal malfunction in which an enzyme called arylsulfatase is deficient. In effect, sulfatides build up in many tissues inside the body which eventually destroys myelin in the nervous system. Symptoms of MLD include:

- Difficulty in walking due to muscle wasting and weakness

- Developmental delays

- Progressive loss of vision leading to blindness

- Convulsions

- Dementia

Currently, there is no known cure or treatment for MLD being used globally, and patients only receive limited management for pain and other symptoms.

Wiskott-Aldrich Syndrome

Wiskott-Aldrich syndrome or WAS is a rare disease characterised by eczema, low platelet count and immune deficiency. Majority of its sufferers are male due to inheritance and first signs includes low platelet counts, nose bleeding and bloody diarrhoea. Other symptoms are:

- Eczema which develops within first month of life

- Recurrent bacterial infections

- Autoimmune disorder

- Lymphoma

- Leukaemia

The treatment for WAS is based on correcting every symptoms by using aspirin and avoidance of other non-steroidal anti-inflammatory drugs. Sometimes, patients undergo blood transfusion and iron supplementation to replace blood loss.

This new therapy using HIV genetic viral vector opens a possible cure or effective treatment for both rare and crippling disease. All of the subjects recovered and finally able to run, play and go to school.

The studies were published in July 11, 2013 in the journal Science.