scispace - formally typeset
M

Maria P. Limberis

Researcher at University of Pennsylvania

Publications -  64
Citations -  2244

Maria P. Limberis is an academic researcher from University of Pennsylvania. The author has contributed to research in topics: Cystic fibrosis & Genetic enhancement. The author has an hindex of 24, co-authored 60 publications receiving 1973 citations. Previous affiliations of Maria P. Limberis include Boston Children's Hospital & Wistar Institute.

Papers
More filters
Journal ArticleDOI

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

Eric W.F.W. Alton, +79 more
TL;DR: In this article, a randomized, double-blind, placebo-controlled, phase 2b trial was conducted to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis, where patients were randomly assigned to receive 5 mL of either nebulised pGM169/GL67A gene-liposome complex or 0·9% saline (placebo) every 28 days for 1 year.
Journal ArticleDOI

The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

TL;DR: Co-instillation of AAV9 vector with neuraminidase into mouse lung resulted in exposure of terminal galactose on the apical surface of conducting airway epithelial cells, as shown by lectin binding and increased transduction of these cells, demonstrating the possible utility of this vector in lung-directed gene transfer.
Journal ArticleDOI

Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro.

TL;DR: It is demonstrated that AAV6.2 exhibits improved transduction efficiency compared to previously reported AAVs in mouse airways and in culture models of human airway epithelium and that this vector requires further development for preclinical and clinical testing.
Journal ArticleDOI

Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered.

TL;DR: It is shown that AAV2/9 can be readministered in the presence of high levels of serum-circulating neutralizing antibodies as early as 1 month after initial exposure, with minimal effect on overall reporter gene expression, rendering it a promising gene transfer vector candidate for use in humans.