Alexandra S. Piotrowski-Daspit
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View article: Systemic in utero gene editing as a treatment for cystic fibrosis
Systemic in utero gene editing as a treatment for cystic fibrosis Open
In utero gene editing has the potential to modify disease-causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a dis…
View article: Cellular determinants influence the red blood cell adsorption efficiency of poly(amine- <i>co</i> -ester) nanoparticles
Cellular determinants influence the red blood cell adsorption efficiency of poly(amine- <i>co</i> -ester) nanoparticles Open
Many poly(amine- co -ester) (PACE) nanoparticles, drug delivery vehicles for nucleic acid and small molecule cargoes, accumulate in the liver and spleen following intravenous administration, limiting delivery to nonhepatosplenic tissues. R…
View article: Systemic in utero gene editing as a treatment for cystic fibrosis
Systemic in utero gene editing as a treatment for cystic fibrosis Open
In utero gene editing has the potential to modify disease causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a dis…
View article: Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys
Enhancing in vivo cell and tissue targeting by modulation of polymer nanoparticles and macrophage decoys Open
The in vivo efficacy of polymeric nanoparticles (NPs) is dependent on their pharmacokinetics, including time in circulation and tissue tropism. Here we explore the structure-function relationships guiding physiological fate of a library of…
View article: Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination
Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination Open
An inhalable platform for messenger RNA (mRNA) therapeutics would enable minimally invasive and lung-targeted delivery for a host of pulmonary diseases. Development of lung-targeted mRNA therapeutics has been limited by poor transfection e…
View article: Rational nanoparticle design: Optimization using insights from experiments and mathematical models
Rational nanoparticle design: Optimization using insights from experiments and mathematical models Open
View article: In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia
In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia Open
View article: Nucleic Acid Delivery to the Vascular Endothelium
Nucleic Acid Delivery to the Vascular Endothelium Open
This Review examines the state-of-the-art in the delivery of nucleic acid therapies that are directed to the vascular endothelium. First, we review the most important homeostatic functions and properties of the vascular endothelium and sum…
View article: In vivo correction of cystic fibrosis mediated by PNA nanoparticles
In vivo correction of cystic fibrosis mediated by PNA nanoparticles Open
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide nucleic acid g…
View article: Inhalable polymer nanoparticles for versatile mRNA delivery and mucosal vaccination
Inhalable polymer nanoparticles for versatile mRNA delivery and mucosal vaccination Open
An inhalable platform for mRNA therapeutics would enable minimally invasive and lung targeted delivery for a host of pulmonary diseases. Development of lung targeted mRNA therapeutics has been limited by poor transfection efficiency and ri…
View article: In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia
In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia Open
Structural fetal diseases, such as congenital diaphragmatic hernia (CDH) can be diagnosed prenatally. Neonates with CDH are healthy in utero as gas exchange is managed by the placenta, but impaired lung function results in critical illness…
View article: <i>In vivo</i> correction of cystic fibrosis mediated by PNA nanoparticles
<i>In vivo</i> correction of cystic fibrosis mediated by PNA nanoparticles Open
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. We sought to correct the multiple organ dysfunction of the F508del CF-causing mutation using systemic delivery of peptide n…
View article: 582: In vivo nanoparticle-mediated therapeutic nucleic acid delivery for CF treatment
582: In vivo nanoparticle-mediated therapeutic nucleic acid delivery for CF treatment Open
View article: Nanoparticle-mediated convection-enhanced delivery of a DNA intercalator to gliomas circumvents temozolomide resistance
Nanoparticle-mediated convection-enhanced delivery of a DNA intercalator to gliomas circumvents temozolomide resistance Open
View article: PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery
PEGylation of poly(amine-co-ester) polyplexes for tunable gene delivery Open
View article: Nanoparticles for delivery of agents to fetal lungs
Nanoparticles for delivery of agents to fetal lungs Open
View article: High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents
High-throughput quantitative microscopy-based half-life measurements of intravenously injected agents Open
Significance The effectiveness of systemically delivered therapeutics is dependent on their residence time in circulation, as this parameter influences the frequency of interaction with intended target sites prior to elimination from the b…
View article: Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo
Poly(Lactic-co-Glycolic Acid) Nanoparticle Delivery of Peptide Nucleic Acids In Vivo Open
View article: Polymeric vehicles for nucleic acid delivery
Polymeric vehicles for nucleic acid delivery Open
View article: Poly(amine-co-ester) nanoparticles for effective Nogo-B knockdown in the liver
Poly(amine-co-ester) nanoparticles for effective Nogo-B knockdown in the liver Open
View article: Nanoparticle-mediated intratumoral inhibition of miR-21 for improved survival in glioblastoma
Nanoparticle-mediated intratumoral inhibition of miR-21 for improved survival in glioblastoma Open
View article: Dynamics of Tissue-Induced Alignment of Fibrous Extracellular Matrix
Dynamics of Tissue-Induced Alignment of Fibrous Extracellular Matrix Open
View article: Interstitial fluid pressure regulates collective invasion in engineered human breast tumors<i>via</i>Snail, vimentin, and E-cadherin
Interstitial fluid pressure regulates collective invasion in engineered human breast tumors<i>via</i>Snail, vimentin, and E-cadherin Open
Many solid tumors exhibit elevated interstitial fluid pressure (IFP). This elevated pressure within the core of the tumor results in outward flow of interstitial fluid to the tumor periphery. We previously found that the directionality of …