First evidence of gene therapy for abnormal blood vessel growth in newborns

July 18, 2001

The first evidence of the potential for gene therapy to treat eye disease that stems from abnormal blood vessel growth is revealed in research published in the British Journal of Ophthalmology.

Gene therapy is a relatively new and much heralded therapeutic approach. But despite the advances in molecular genetics, attempts to target organs or particular body structures using the technique have proved disappointing. The problem appears to hinge on the vector agent that is used to target cellular DNA. It must be capable of successfully delivering a genetic sequence and at the same time be able to withstand the receiving body's immune system response.

The researchers studied newborn rats with induced retinopathy of prematurity. This is a disease which is on the increase as a result of the rising numbers of surviving premature babies. It is a leading cause of blindness and defective vision in children, and despite surgical advances, around half of babies with the most severe form of the disease will become blind. Disordered blood vessel growth from the retina needs to be prevented in order to treat the disease.

The researchers tested the ability of four different viruses, adenovirus, bovine smallpox virus, herpes virus, and a virus that causes leukaemia, the murine leukaemia virus, to transport genetic material into the defective eyes of 18 day old rats.

The results showed that adenovirus was the most effective vector, and furthermore it specifically targeted tissues in the jelly-like material inside the eyeball which destroy vital developing eye tissues. Delicate adjacent nerve tissues risk being damaged if the target is not specific.

The research is in its early stages, say the authors, but they suggest that this is a first step in the use of gene therapy for both treatment and research purposes.

Dr Itay Chowers, Department of Ophthalmology, Hadassah University Hospital, Hadassah, Israel.
Tel: 001-410-502-5230 (USA)

Dr William Good, Smith-Kettlewell Eye Research Institute, San Francisco, USA.
Tel: 001-415-345-2000

BMJ Specialty Journals

Related Gene Therapy Articles from Brightsurf:

Risk of AAV mobilization in gene therapy
New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy.

Discovery challenges the foundations of gene therapy
An article published today in Science Translational Medicine by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the liver.

Gene therapy: Novel targets come into view
Retinitis pigmentosa is the most prevalent form of congenital blindness.

Gene therapy targets inner retina to combat blindness
Batten disease is a group of fatal, inherited lysosomal storage disorders that predominantly affect children.

New Human Gene Therapy editorial: Concern following gene therapy adverse events
Response to the recent report of the deaths of two children receiving high doses of a gene therapy vector (AAV8) in a Phase I trial for X-linked myotubular myopathy (MTM).

Restoring vision by gene therapy
Latest scientific findings give hope for people with incurable retinal degeneration.

Gene therapy/gene editing combo could offer hope for some genetic disorders
A hybrid approach that combines elements of gene therapy with gene editing converted an experimental model of a rare genetic disease into a milder form, significantly enhancing survival, shows a multi-institutional study led by the University of Pennsylvania and Children's National Hospital in Washington, D.C.

New technology allows control of gene therapy doses
Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing.

Gene therapy: Development of new DNA transporters
Scientists at the Institute of Pharmacy at Martin Luther University Halle-Wittenberg (MLU) have developed new delivery vehicles for future gene therapies.

Gene therapy promotes nerve regeneration
Researchers from the Netherlands Institute for Neuroscience and the Leiden University Medical Center have shown that treatment using gene therapy leads to a faster recovery after nerve damage.

Read More: Gene Therapy News and Gene Therapy Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to