S
S H Cheng
Researcher at Genzyme
Publications - 42
Citations - 2020
S H Cheng is an academic researcher from Genzyme. The author has contributed to research in topics: Genetic transfer & Cystic fibrosis transmembrane conductance regulator. The author has an hindex of 25, co-authored 41 publications receiving 1978 citations.
Papers
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Journal ArticleDOI
Maturation and function of cystic fibrosis transmembrane conductance regulator variants bearing mutations in putative nucleotide-binding domains 1 and 2.
Richard J. Gregory,D. P. Rich,S H Cheng,David W. Souza,Sucharita Paul,Parthasarathy Manavalan,Matthew P. Anderson,M. J. Welsh,Alan E. Smith +8 more
TL;DR: The results show that all CF-associated mutants, with one exception, lack functional activity as measured in the SPQ assay, and that the structure of NBD1 itself or of the surrounding domains renders it particularly sensitive to mutational changes.
Journal ArticleDOI
Comparison between cationic polymers and lipids in mediating systemic gene delivery to the lungs.
Alessandra Bragonzi,Alessandra Boletta,A. Biffi,Anna Muggia,Giovanna Sersale,S H Cheng,Claudio Bordignon,Baroukh M. Assael,Massimo Conese +8 more
TL;DR: A nonviral approach for transfecting various airway-derived cell lines in vitro showed that cationic polymers and lipids are able to transfect with high efficiency the reporter genes firefly luciferase and E. coli lacZ.
Journal ArticleDOI
Functional activation of the cystic fibrosis trafficking mutant delta F508-CFTR by overexpression
S H Cheng,Shaona Lee Fang,Joseph Zabner,John Marshall,S. Piraino,S. C. Schiavi,Douglas M. Jefferson,M. J. Welsh,Alan E. Smith +8 more
TL;DR: The observation that overexpression can effect the presence of recombinant delta F508-CFTR at the plasma membrane suggests that perhaps other butyrate-like compounds that are more potent and more specific for the promoter of the CF gene may be efficacious in alleviating the Cl- channel defect associated with CF.
Journal ArticleDOI
The extra- and intracellular barriers to lipid and adenovirus-mediated pulmonary gene transfer in native sheep airway epithelium.
C Kitson,B Angel,D Judd,Stephen Rothery,Nicholas J. Severs,Ann Dewar,Leaf Huang,S C Wadsworth,S H Cheng,Duncan M. Geddes,Eric W.F.W. Alton +10 more
TL;DR: An ex vivo model which retains many of the characteristics of in vivo native airways including mucociliary clearance, mucus coverage and an intact cellular structure is developed and several barriers to gene transfer are demonstrated.
Journal ArticleDOI
Gene therapy progress and prospects: gene therapy of lysosomal storage disorders.
S H Cheng,Alan E. Smith +1 more
TL;DR: This review will highlight the progress that has been made over the past 2 years in preclinical research for this group of disorders and speculate on future prospects.