Gernutik lortutako zelula ama mesenkimalak (hUSC) pseudohipoparatiroidismoaren (PHP) terapia geniko ez-biralerako

Abstract

Zelula ama mesenkimalak (MSC) giza gorputzean hainbat nitxotan aurkitzen diren zelula ama heldu multipotenteak dira. Gizakien gernutik lortutako MSCak (hUSC), ohiko MSCen abantaila biologiko guztiak mantentzen dituzte eta era ez-inbaditzailean lortzen dira. Pseudohipoparatiroidismoa (PHP) gaixotasun (epi)genetiko talde heterogeneo eta arraroa da, hormona paratiroideoaren (PTH) aurkako erresistentziak ezaugarritzen dituena. Ikerketa honetan hUSCak isolatzeko protokoloa garatu dugu eta niosoma deritzan bektore ez-biral lipidikoekin transfektatzeko prozedura zehaztasunez aztertu dugu, etorkizunean PHP moduko gaixotasunen terapia genikoan aplikatu ahal izateko.

Mesenchymal stem cells (MSCs) are adult multipotent stem cells that can be found in several niches. Human urine-derived mesenchymal stem cells (hUSCs) maintain the advantages of MSCs and in addition, they can be isolated in a non-invasive way from and easily accessible source. Pseudohypoparathyroidism (PHP) is an heterogeneous group of (epi)genetic disorders characterized by parathyroid hormone (PTH) resistance. In this research, we have developed and validated a protocol based on the non-viral vectors known as niosomes for transfecting hUSCs. This strategy could be used in the future with gene therapy purposes in genetic diseases such as PHP.