Related references
Note: Only part of the references are listed.Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles
Stephanie Lorain et al.
MOLECULAR THERAPY (2008)
Local dystrophin restoration with antisense oligonucleotide PRO051
Judith C. van Deutekom et al.
NEW ENGLAND JOURNAL OF MEDICINE (2007)
Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice
Rachid Benchaouir et al.
CELL STEM CELL (2007)
Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications
Annemieke Aartsma-Rus et al.
RNA (2007)
Morpholino oligomer -: Mediated Exon skipping averts the onset of dystrophic pathology in the mdx mouse
Sue Fletcher et al.
MOLECULAR THERAPY (2007)
Dystrophin-deficient mdx mice display a reduced life span and are susceptible to spontaneous rhabdomyosarcoma
Jeffrey S. Chamberlain et al.
FASEB JOURNAL (2007)
Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression
Zejing Wang et al.
MOLECULAR THERAPY (2007)
Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
G. McClorey et al.
GENE THERAPY (2006)
Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice
Michela Alessandra Denti et al.
HUMAN GENE THERAPY (2006)
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model
MA Denti et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
J Alter et al.
NATURE MEDICINE (2006)
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucteotide
S Fletcher et al.
JOURNAL OF GENE MEDICINE (2006)
Long-term pharmacologically regulated expression of erythropoietin in,primates following AAV-mediated gene transfer
VM Rivera et al.
BLOOD (2005)
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
QL Lu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Targeted exon skipping in transgenic hDMD mice:: A model for direct preclinical screening of human-specific antisense oligonucleotides
M Bremmer-Bout et al.
MOLECULAR THERAPY (2004)
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
A Goyenvalle et al.
SCIENCE (2004)
Advances in Duchenne muscular dystrophy gene therapy
JCT van Deutekom et al.
NATURE REVIEWS GENETICS (2003)
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
QL Lu et al.
NATURE MEDICINE (2003)
U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping
C Brun et al.
CELLULAR AND MOLECULAR LIFE SCIENCES (2003)
Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy
CJ Mann et al.
JOURNAL OF GENE MEDICINE (2002)
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells
FG De Angelis et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model
A Auricchio et al.
HUMAN MOLECULAR GENETICS (2001)
Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells
JCT van Deutekom et al.
HUMAN MOLECULAR GENETICS (2001)
Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column
A Auricchio et al.
HUMAN GENE THERAPY (2001)
Comparative evolution of muscular dystrophy in diaphragm, gastrocnemius and masseter muscles from old male mdx mice
J Muller et al.
JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY (2001)
Share Paper
