Search Results - "Dave, Apeksha"
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In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
Published in Nature communications (13-07-2021)“…In utero base editing has the potential to correct disease-causing mutations before the onset of pathology. Mucopolysaccharidosis type I (MPS-IH, Hurler…”
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Small RNA combination therapy for lung cancer
Published in Proceedings of the National Academy of Sciences - PNAS (26-08-2014)“…MicroRNAs (miRNAs) and siRNAs have enormous potential as cancer therapeutics, but their effective delivery to most solid tumors has been difficult. Here, we…”
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Disparities in Stage-Specific Guideline-Concordant Cancer-Directed Treatment for Patients with Pancreatic Adenocarcinoma
Published in Journal of gastrointestinal surgery (01-11-2021)“…Background The utilization of cancer-directed treatment for patients with all stages of pancreatic cancer in the USA is unknown. This study sought to examine…”
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Evaluating the ACS NSQIP Risk Calculator in Primary Pancreatic Neuroendocrine Tumor: Results from the US Neuroendocrine Tumor Study Group
Published in Journal of gastrointestinal surgery (01-11-2019)“…Background In a changing health care environment where patient outcomes will be more closely scrutinized, the ability to predict surgical complications is…”
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Sustained Engraftment in Fanconi Anemia (FA) Mice Using in Utero Hematopoietic Cell Transplantation (IUHCT)
Published in Blood (15-11-2022)Get full text
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Maternal Cas9 Antibodies Do Not Prevent Fetal Gene Editing
Published in Journal of the American College of Surgeons (01-05-2023)Get full text
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Targeting of Fetal and Neonatal Pulmonary Epithelial Cells for Therapeutic Applications
Published in Journal of the American College of Surgeons (01-05-2023)Get full text
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Fetal Pulmonary Genome Modification via Direct Intratracheal Injection in the Mouse
Published in Journal of the American College of Surgeons (01-11-2021)Get full text
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Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery
Published in Journal of controlled release (01-01-2022)“…Congenital disorders resulting in pathological protein deficiencies are most often treated postnatally with protein or enzyme replacement therapies. However,…”
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In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight
Published in Nature nanotechnology (01-08-2014)“…Dysfunctional endothelium contributes to more diseases than any other tissue in the body. Small interfering RNAs (siRNAs) can help in the study and treatment…”
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Intracranial Delivery of Lipid Nanoparticles in the Murine Model
Published in Journal of the American College of Surgeons (01-11-2022)Get full text
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The Extrauterine Environment for Neonatal Development Supports Mid-Gestation Fetal Lambs
Published in Journal of the American College of Surgeons (01-11-2022)Get full text
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Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease
Published in ACS nano (25-07-2023)“…Delivery of mRNA-based therapeutics to the perinatal brain holds great potential in treating congenital brain diseases. However, nonviral delivery platforms…”
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Biomimetic and synthetic esophageal tissue engineering
Published in Biomaterials (01-07-2015)“…Abstract Background/Purpose A tissue-engineered esophagus offers an alternative for the treatment of pediatric patients suffering from severe esophageal…”
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National Congenital Diaphragmatic Hernia Outcomes: Assessing Volume-Outcomes Relationships Using the 2016 KID Database
Published in Journal of the American College of Surgeons (01-11-2021)Get full text
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Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in mice
Published in The Journal of clinical investigation (15-06-2024)“…In utero gene editing (IUGE) is a potential treatment for inherited diseases that cause pathology before or soon after birth. Preexisting immunity to…”
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Assessment of extremely premature lambs supported by the Extrauterine Environment for Neonatal Development (EXTEND)
Published in Pediatric research (04-06-2024)“…Our team has previously reported physiologic support by the EXTra-uterine Environment for Neonatal Development (EXTEND) of 105 to 117 days gestational age (GA)…”
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Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
Published in Gene therapy (01-04-2023)Get full text
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In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells
Published in Proceedings of the National Academy of Sciences - PNAS (06-08-2024)“…Monogenic blood diseases are among the most common genetic disorders worldwide. These diseases result in significant pediatric and adult morbidity, and some…”
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