Background

1. Since gene therapy was first attempted in1989, there have been over 300 clinical protocols approved world wide. 30 research trials in patients have been considered in the UK by the Gene Therapy Advisory Committee (GTAC) established in 1993 to review such work [1].

2. There is a wide consensus that, at present, gene therapy should be restricted to attempts to modify somatic (body) cells so that changes will not be passed to successive generations. GTAC has confirmed that its view remains that gene modification of the germ line, where effects could be transmitted to offspring, should not yet be attempted.

3. To date, all but two of the trials of somatic gene therapy considered by GTAC have been intended to be performed in adults or young persons over 16 years. The two exceptions were in young children born with inherited single gene disorders, Hurlers Disease and Severe Combined Immuno-Deficiency (SCID), where it may be possible to correct the gene deficiency before serious or life threatening symptoms develop.

4. However in many genetic disorders it may not be possible to use such potential therapies after birth. In some disorders the damage is already done before birth and there may be good clinical reasons to intervene in utero to try and correct the genetic damage.

5. Interventions in utero are not new. Surgical procedures are used to correct the accumulation of fluid in the bladder or chest; steroid drugs can be infused into the developing fetus and transfusion of blood or platelets directly into the fetus are established procedures.

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