F508-del CFTR rescue by mutated toxoids and evaluation of its functional efficacy in vivo

Many genetic diseases caused by small genetic mutations do not result in complete loss of protein fnction, but rather are due to the degradation of mutant protein by a “quality control” mechanism (endoplasmic reticulum-associated degradation-ERAD) soon after it is made. Prominent amongst such ERAD-dependent diseases is Cystic Fibrosis(CF), a chronic, progressive, life-threatening genetic disease that […]

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