We attempted to find some compounds for the effective delivery of gene constructs into cells and obtained two trispherical dendrimers on the basis of lysine, (Lys)8-(alpha, epsilon-Lys)4-(alpha, epsilon-Lys)2-(alpha, epsilon-Lys)-Ala-NH2 (D1) and (Lys)8-(alpha, epsilon-Lys)4-(alpha, epsilon-Lys)2-(alpha, epsilon-Lys)-Ala-[Lys(Plm)]2-Ala-NH2 (D2), as well as the starburst polymeric derivatives of D1, (pVIm)8-D1 and (pLys)n-D1, containing poly(N-vinylimidazole) and polylysine chains bound at a single point to the dendrimer amino groups. The conditions of dendrimer-plasmid DNA complex formation were studied. The intracellular localization of these complexes and the expression of gene constructs delivered with their help were analyzed in transfection experiments on the HeLa cell cultures of human epithelial carcinoma and on C2C12 mouse myoblasts. It was found that the chemical structure of dendrimer D1 and its derivatives significantly affected the structure and properties of complex. The English version of the paper: Russian Journal of Bioorganic Chemistry, 2004, vol. 30, no. 1; see also http://www.maik.ru.

Patterns of dystrophin and ␤-galactosidase expression ticles were detected by FISH analysis in about 60-70% of were examined in mdx mice after i.m. injections of synmyofiber nuclei in muscles of injected and contralateral thetic microspheres (MF-2) loaded with full-length limbs 7 days after application. The presence of human dys-(pHSADy) or mini-dystrophin gene (pSG5dys) cDNA plastrophin cDNA and its products in all skeletal muscles and mid constructs or with LacZ marker gene (pCMV-LacZ). A in different internal organs was proven by PCR and RTsingle injection of 25 g pHSADy into quadriceps femoris PCR analysis. Patches of ␤-galactosidase expression were muscle resulted in 6.8% of dystrophin positive myofibers abundant in injected muscle, and frequent in the contralat-(DPM) in a given muscle; 8.4% of DPM in glutaeus muscle eral and other skeletal muscles as well as in diaphragm, and 4.3% of DPM in quadriceps femoris muscle of contraheart and lungs. High levels of dystrophin cDNA lateral limb on day 21 after exposure compared with only expression, and an efficient distant transfection effect with 0.6% DPM in intact (non-injected) mdx mice. A high propreferential intranuclei inclusion of MF-2 vehicle, are very portion of DPM (17.6% and 10.8%, respectively) was regisencouraging for the development of a new constructive tered in both injected and contralateral muscles after ministrategy in gene therapy trials of DMD. gene cDNA administration. MF-2/dystrophin cDNA par-