Gene Therapy Update


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Dr Pamela Becker Assistant Professor at the University of Massachusetts Medical Center, has been carrying out research into gene therapy for Gauchers disease since March 1996 and has collaborated with Prof John Barranger on the gene therapy trial he carried out on three patients with Gauchers disease. She spoke about the trial at the Gauchers Conference in Memphis.

Dr Becker reported that a fourth patient has started on the gene therapy trial receiving a second gene transfer that week. The patient is scheduled to have four gene transfers in all at three monthly intervals. Another two patients are due to start shortly.

Modifications have been made to the process for transferring the new healthy gene into the patientµs blood stem cells which is hoped will improve the amount of new gene introduced and therefore produce more enzyme.

'It does not matter what the genotype (inherited genetic mutations) of the patient is. The hope is to raise the enzyme activity from its deficient level. It may be that the bone marrow environment needs to be good to enable the modified stem cells to engraft (attach themselves).

'A lot of work still needs to be done but I hope that gene therapy will be an effective treatment in 5-10 years .µ

Dr Becker said that every person has about 100,000 genes and anyone of these can cause illness. Since five years ago, several thousand patients suffering from different diseases have undergone gene therapy although results have so far not been effective. Over two thirds have been on cancer trials and only 0.2% on congenital diseases such as Gauchers disease.


Medical Management of Gauchers Disease

My Aching Bones and Fosamax

Gene Therapy

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Source: Gauchers News January 1999

© Copyright Gauchers Association 1999