• Publications
  • Influence
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
  • B. Wu, H. Moulton, +11 authors Q. Lu
  • Biology, Medicine
  • Proceedings of the National Academy of Sciences
  • 30 September 2008
Antisense oligonucleotide-mediated exon skipping is able to correct out-of-frame mutations in Duchenne muscular dystrophy and restore truncated yet functional dystrophins. However, its application isExpand
  • 220
  • 6
Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.
Steric-block antisense oligonucleotides (AONs) are able to target RNAs for destruction and splicing alteration. Reading frame restoration of the dystrophin transcript can be achieved by AON-mediatedExpand
  • 149
  • 5
One-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.
  • B. Wu, B. Xiao, +7 authors Q. Lu
  • Biology, Medicine
  • Molecular therapy : the journal of the American…
  • 1 March 2011
Antisense therapy has been successful to skip targeted dystrophin exon with correction of frameshift and nonsense mutations of Duchenne muscular dystrophy (DMD). Systemic production of truncated butExpand
  • 60
  • 3
Targeted Skipping of Human Dystrophin Exons in Transgenic Mouse Model Systemically for Antisense Drug Development
Antisense therapy has recently been demonstrated with great potential for targeted exon skipping and restoration of dystrophin production in cultured muscle cells and in muscles of Duchenne MuscularExpand
  • 24
  • 2
  • PDF
Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.
  • B. Wu, P. Lu, +6 authors Q. Lu
  • Biology, Medicine
  • The American journal of pathology
  • 1 August 2012
Exon skipping is capable of correcting frameshift and nonsense mutations in Duchenne muscular dystrophy. Phase 2 clinical trials in the United Kingdom and the Netherlands have reported induction ofExpand
  • 62
  • 1
High efficiency and low toxicity of polyethyleneimine modified Pluronics (PEI–Pluronic) as gene delivery carriers in cell culture and dystrophic mdx mice
A series of low molecular weight polyethyleneimine (LPEI) conjugated Pluronic copolymers (PCMs) were synthesized and evaluated in C2C12 myoblasts and CHO cells in vitro and in dystrophic mdx mice inExpand
  • 28
  • 1
  • PDF
Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.
  • Yihong Hu, B. Wu, +7 authors Q. Lu
  • Biology, Medicine
  • Molecular therapy : the journal of the American…
  • 1 April 2010
Exon skipping has demonstrated great potential for treating Duchenne muscular dystrophy (DMD) and other diseases. We have developed a drug-screening system using C2C12 myoblasts expressing a reporterExpand
  • 25
  • 1
Glucocorticoid Steroid and Alendronate Treatment Alleviates Dystrophic Phenotype with Enhanced Functional Glycosylation of α-Dystroglycan in Mouse Model of Limb-Girdle Muscular Dystrophy with
  • B. Wu, S. Shah, +10 authors Q. Lu
  • Medicine
  • The American journal of pathology
  • 1 June 2016
Fukutin-related protein-muscular dystrophy is characterized by defects in glycosylation of α-dystroglycan with variable clinical phenotypes, most commonly as limb-girdle muscular dystrophy 2I. ThereExpand
  • 12
  • 1
  • PDF
Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene
Loss-of-function mutations in the Fukutin-related protein (FKRP) gene cause limb-girdle muscular dystrophy type 2I (LGMD2I) and other forms of congenital muscular dystrophy-dystroglycanopathy thatExpand
  • 15
  • 1
Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice
Antisense therapy with both chemistries of phosphorodiamidate morpholino oligomers (PMOs) and 2′-O-methyl phosphorothioate has demonstrated the capability to induce dystrophin expression in DuchenneExpand
  • 30