C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/89 (2006.01) A01K 67/027 (2006.01) A61K 35/52 (2006.01) A61K 48/00 (2006.01) C12N 5/10 (2006.01) C12N 15/86 (2006.01) C12N 15/87 (2006.01) C12N 5/076 (2010.01)
Patent
CA 2309904
A composition for in vivo transfection of vertebrate male germ cells comprises a nucleic acid or transgene, and a gene delivery system, and optionally a protective internalizing agent, such as an endosomal lytic agent, a virus or a viral component, which is internalized by cells along with the transgene and which enhances gene transfer through the cytoplasm to the nucleus of the male germ cell. A pharmaceutical preparation and a transfer kit utilize the composition. A method for introducing a polynucleotide into vertebrate male germ cells comprises the administration of the composition to a vertebrate. A method for isolating or selecting transfected cells utilizes a reporter gene, and a method for administering transfected male germ cells utilizes male germ cells which have been transfected in vitro.
L'invention concerne une composition destinée à la transfection in vivo de cellules germinales mâles de vertébrés comprenant un acide nucléique ou un transgène, un système d'apport génique, et éventuellement un agent d'internalisation protecteur, tel qu'un agent lytique endosomique, un virus ou un composant viral, qui est internalisé par des cellules ainsi que par les transgènes et qui améliore le transfert génique à travers le cytoplasme vers le noyau de la cellule germinale mâle. Une préparation pharmaceutique et un kit de transfert utilisent cette composition. Une méthode d'introduction d'un polynucléotide dans des cellules germinales mâles de vertébrés consiste à administrer cette composition à un vertébré. Une méthode d'isolement ou de sélection de cellules transfectées utilise un gène rapporteur, et une méthode d'administration de cellules germinales mâles transfectées utilise des cellules germinales mâles ayant été transfectées in vitro.
Hovatta Outi
Readhead Carol W.
Winston Robert
Cedars-Sinai Medical Center
Gowling Lafleur Henderson Llp
Imperial College Innovations Ltd.
Imperial College Of Science Technology And Medicine
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