Publications Poster: Intratracheal administration of AAV2.5T-SP183-fCFTRΔR in combination with doxorubicincorrects the mucociliary clearance defect in cystic fibrosis model ferrets
Publications SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis
Publications Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRDR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets
Publications Repeat Dosing of AAV2.5T to Ferret Lungs Elicits an Antibody Response That Diminishes Transduction in an Age-Dependent Manner
Publications Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell
Publications Inhalation of SP-101 followed by doxorubicin results in wide-spread SP-101 distribution and hCFTRΔR transgene expression in the airways of CF and non-CF ferrets
Publications SP-101 gene therapy restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of CF and non-CF ferrets
Publications Immunosuppression reduces rAAV2.5T neutralizing antibodies that limit efficacy following repeat dosing to ferret lungs
Publications Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRDR gene delivery to adult ferret lungs
Publications Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferrets
Publications Poster: 621: Administration of SP-101 and doxorubicin results in robust and durable human cystic fibrosis transmembrane conductance regulator minigene transgene expression in the airways of ferrets and corrects human cystic fibrosis airway epithelia in vitro. North American Cystic Fibrosis Conference 2022
Publications Poster: 672: SP-101 activity increases in a dose dependent manner to both vector and doxorubicin dose. North American Cystic Fibrosis Conference 2022
Publications Abstract: 672: SP-101 activity increases in a dose dependent manner to both vector and doxorubicin dose. North American Cystic Fibrosis Conference 2022
Publications Abstract: 621: Administration of SP-101 and doxorubicin results in robust and durable human cystic fibrosis transmembrane conductance regulator minigene transgene expression in the airways of ferrets and corrects human cystic fibrosis airway epithelia in vitro. North American Cystic Fibrosis Conference 2022
Publications Katherine Excoffon: Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferrets. North American Cystic Fibrosis Conference 2021. – Poster
Publications Katherine Excoffon: Delivery of SP-101 restores CFTR function in human CF airway epithelial cultures and drives hCFTRΔR transgene expression in the airways of ferrets. North American Cystic Fibrosis Conference 2021. – Video
Publications Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers
