Advances in Duchenne muscular dystrophy gene therapy
JCT Van Deutekom, GJB Van Ommen - Nature Reviews Genetics, 2003 - nature.com
Since the initial characterization of the genetic defect for Duchenne muscular dystrophy,
much effort has been expended in attempts to develop a therapy for this devastating …
much effort has been expended in attempts to develop a therapy for this devastating …
Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy
T Koo, MJ Wood - Human gene therapy, 2013 - liebertpub.com
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by
mutations in the DMD gene, affecting 1 in 3500 newborn males. Complete loss of muscle …
mutations in the DMD gene, affecting 1 in 3500 newborn males. Complete loss of muscle …
The FSHD atrophic myotube phenotype is caused by DUX4 expression
C Vanderplanck, E Ansseau, S Charron, N Stricwant… - PloS one, 2011 - journals.plos.org
Background Facioscapulohumeral muscular dystrophy (FSHD) is linked to deletions in 4q35
within the D4Z4 repeat array in which we identified the dou ble homeobo x 4 (DUX4) gene …
within the D4Z4 repeat array in which we identified the dou ble homeobo x 4 (DUX4) gene …
Morpholino antisense oligonucleotide induced dystrophin exon 23 skip** in mdx mouse muscle
The mdx mouse model of muscular dystrophy arose due to a nonsense mutation in exon 23
of the dystrophin gene. We have previously demonstrated that 2′-O-methyl …
of the dystrophin gene. We have previously demonstrated that 2′-O-methyl …
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide
S Fletcher, K Honeyman, AM Fall… - The Journal of Gene …, 2006 - Wiley Online Library
Abstract Background Duchenne and Becker muscular dystrophies are allelic disorders
arising from mutations in the dystrophin gene. Duchenne muscular dystrophy is …
arising from mutations in the dystrophin gene. Duchenne muscular dystrophy is …
Functional analysis of 114 exon-internal AONs for targeted DMD exon skip**: indication for steric hindrance of SR protein binding sites
A Aartsma-Rus, CL De Winter, AAM Janson… - …, 2005 - liebertpub.com
As small molecule drugs for Duchenne muscular dystrophy (DMD), antisense
oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD …
oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD …
Multiple exon skip** compositions for DMD
P Sazani, R Kole - US Patent 8,871,918, 2014 - Google Patents
US8871918B2 - Multiple exon skip** compositions for DMD - Google Patents US8871918B2
- Multiple exon skip** compositions for DMD - Google Patents Multiple exon skip** …
- Multiple exon skip** compositions for DMD - Google Patents Multiple exon skip** …
Compositions for treating muscular dystrophy
EM Kaye - US Patent 9,506,058, 2016 - Google Patents
2020-01-09 Assigned to BIOPHARMA CREDIT PLC reassignment BIOPHARMA CREDIT
PLC CORRECTIVE ASSIGNMENT TO CORRECT THE TYPOGRAPHICAL ERROR APP. NO …
PLC CORRECTIVE ASSIGNMENT TO CORRECT THE TYPOGRAPHICAL ERROR APP. NO …
The influence of antisense oligonucleotide length on dystrophin exon skip**
PL Harding, AM Fall, K Honeyman, S Fletcher… - Molecular Therapy, 2007 - cell.com
Antisense oligonucleotides (AOs) can be used to redirect dystrophin pre-messenger RNA
(mRNA) processing, to remove selected exons from the mature dystrophin mRNA, to …
(mRNA) processing, to remove selected exons from the mature dystrophin mRNA, to …
Targeted exon skip** in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides
M Bremmer-Bout, A Aartsma-Rus, EJ De Meijer… - Molecular Therapy, 2004 - cell.com
The therapeutic potential of frame-restoring exon skip** by antisense oligonucleotides
(AONs) has recently been demonstrated in cultured muscle cells from a series of Duchenne …
(AONs) has recently been demonstrated in cultured muscle cells from a series of Duchenne …