C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 5/10 (2006.01) A61K 35/12 (2006.01) C12N 5/06 (2006.01) C12N 15/90 (2006.01)
Patent
CA 2535816
The invention relates to various methods of treating lysosomal storage disorders using somatic cells and methods of delivering therapeutic enzymes. More particularly, gain-of-function or loss-of-function mutations in components of the intracellular Golgi to lysosome sorting pathway are used to enhance secretion of one or more lysosomal enzymes in somatic cells, thereby providing treatment for lysosomal storage disorders, particularly in neuronal cells. In addition, homologous recombination may be used to engineer therapeutically useful dells, for example, somatic cells, such as, glial progenitor cells, mesenchymal stem cells and astrocyte precursor cells, to enhance secretion of one or more lysosomal enzymes.
La présente invention concerne, d'une part différents procédés permettant de traiter les troubles lysosomiaux en utilisant des cellules somatiques, et d'autre part des procédés permettant l'apport d'enzymes thérapeutiques. En l'occurrence, des mutations à gain ou perte de fonctions affectant des composants des corps de Golgi intracellulaires intervenant dans le chemin de tri des lysosomes servant à renforcer la sécrétion d'une ou plusieurs enzymes lysosomales dans ces cellules neuronales. En outre, la recombinaison homologue peut servir à la production par génie génétique de cellules thérapeutiquement utiles, par exemple des cellules somatiques telles que des cellules gliales progénitrices, des cellules souches du mésenchyme, et des cellules précurseurs des astrocytes, pour renforcer la sécrétion d'une ou plusieurs enzymes lysosomales.
Dimock Stratton Llp
Q. Therapeutics Inc.
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