Stephen Hart
0000-0001-8254-376X
182 papers found
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Lipid Nanoparticles Efficiently Deliver the Base Editor ABE8e for COL7A1 Correction in Dystrophic Epidermolysis Bullosa Fibroblasts In Vitro
Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing
BMI1 Transduction of Human Airway Epithelial Cells for Expansion of Proliferation and Differentiation
Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR
siRNA silencing of cellular entry proteins as a gene therapy strategy for Covid-19
Lipid-peptide nanocomplexes for mRNA delivery in vitro and in vivo
Integrin‐Targeted, Short Interfering RNA Nanocomplexes for Neuroblastoma Tumor‐Specific Delivery Achieve MYCN Silencing with Improved Survival
Design and Optimisation of Synovial-Targeted Non-Viral Lipid Nanoparticles for Systemic Delivery in Rheumatoid Arthritis
Higher throughput drug screening for rare respiratory diseases: readthrough therapy in primary ciliary dyskinesia
CRISPR/Cas9 Gene Editing Therapies for Cystic Fibrosis
Assembly strategy of liposome and polymer systems for siRNA delivery
The liposomal delivery of hydrophobic oxidovanadium complexes imparts highly effective cytotoxicity and differentiating capacity in neuroblastoma tumour cells
MYCN Silencing by RNAi Induces Neurogenesis and Suppresses Proliferation in Models of Neuroblastoma with Resistance to Retinoic Acid
Allele-Specific Small Interfering RNA Corrects Aberrant Cellular Phenotype in Keratitis-Ichthyosis-Deafness Syndrome Keratinocytes
Targeted Non-Viral Delivery of Mini-Intronic Plasmids for Rheumatoid Arthritis
ENaC Targeted Therapy for Cystic Fibrosis
Toward gene therapy in rheumatoid arthritis
New approaches to genetic therapies for cystic fibrosis
Silencing E3 Ubiqutin ligase ITCH as a potential therapy to enhance chemotherapy efficacy in p53 mutant neuroblastoma cells
Liposomal delivery of hydrophobic RAMBAs provides good bioavailability and significant enhancement of retinoic acid signalling in neuroblastoma tumour cells
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