Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy.
Mary Ann Liebert, Inc.Sep 9 2024 Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus gene therapy for the treatment of cystic fibrosis in the peer-reviewed journal Human Gene Therapy . They also showed that after single dose inhaled delivery of SP-101, SP-101 vector genomes were detected throughout the respiratory tract of CF ferrets.
The investigators showed that hCFTRΔR mRNA expression was highest in ferrets that also received single does inhaled doxorubicin, an AAV transduction augmenter. In in vitro studies, "functional correction of CF human airway epithelia increased with increasing multiplicity of infection and doxorubicin concentration and correlated with increasing cell-associated vector genomes and hCFTRΔR mRNA expression."
Fibrosis Gene Gene Therapy Cell Doxorubicin Education Genetic Medical School Research Respiratory Virus
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