Compsn. for gene therapy, esp. of viral infections and tumours - comprises anti-sense DNA or RNA inserted into long terminal repeat or transposable element and e.g. virus envelope as carrier

摘要

Compsn. comprises (1) antisense viral RNA and/or viral or oncogene DNA provided with suitable promoters and incorporated into long terminal repeats; (2) a virus envelope, suitable both for the size of the antisense genome which is to be transferred into cells and for specific uptake into target cells; and (3) enzymes and chemicals to assist integration of the antisense nucleic acid. Alternatively, the nucleic acid component of (1) is formed as a transposable element and/or the virus envelope in (2) is replaced by receptor-specific substances to which the antisense nucleic acid is bonded. USE/ADVANTAGE - The compsn. is used to inhibit replication of viruses and tumour genes, for treatment or prevention and is practically free of side effects. A partic. application is treatment of AIDS, where control of sec. infections can also be provided (since the nucleic acid may be derived from many different viruses to impart multiple specificities). Prenatal immunisation against retroviruses is possible and immunity will be transferred to offspring. The compsn. is unaffected by most viral mutations since small variations in the sequence are not enough to prevent hybridisation. It can be produced quickly and in large amounts, and the same process is applicable to all viruses.