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Before gene therapy can become a permanent cure for a condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be stable. Problems with integrating therapeutic DNA into the genome and the rapidly dividing nature of many cells prevent it from achieving _______________. So patients require multiple treatments.

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If the DNA is integrated in a sensitive spot in the genome, for example in a tumor suppressor gene, the therapy could induce a tumor. This has occurred in clinical trials for X-linked severe combined immunodeficiency (X-SCID) patients, in which hematopoietic stem cells were transduced with a corrective transgene using a retrovirus, and this led to the development of _______________ in 3 of 20 patients.

By _______, the FDA approved Genetech's first successful product, a synthetic form of human insulin produced by bacteria that were engineered to contain the insulin gene.

The first gene therapy for an inherited _____ disease was reported by three independent clinical trials in 2008 in patients with Leber's congenital amaurosis (LCA), an early onset retinal dystrophy.

Leber's congenital amaurosis (LCA), an early onset retinal dystrophy, is caused by mutations in retinal pigment epithelium-specific protein 65 kDa (RPE65) gene that encodes a _____________________.