Allele-specific Col1a1 silencing reduces mutant collagen in fibroblasts from Brtl mouse, a model for classical osteogenesis imperfecta

Allele dependent silencing of COL1A2 using small interfering RNAs

(2012) Katarina Lindahl et al.   International Journal of Medical Sciences

Sclerostin antibody improves skeletal parameters in a Brtl/+ mouse model of osteogenesis imperfecta

(2012) Benjamin P Sinder et al.   JOURNAL OF BONE AND MINERAL RESEARCH

Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy

(2012) Ryan E. Loy et al.   PLoS One

Impaired Osteoblastogenesis in a Murine Model of Dominant Osteogenesis Imperfecta: A New Target for Osteogenesis Imperfecta Pharmacological Therapy

(2012) Roberta Gioia et al.   STEM CELLS

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

Progress Toward In Vivo Use of siRNAs-II

(2011) Garrett R Rettig et al.   MOLECULAR THERAPY

New perspectives on osteogenesis imperfecta

(2011) Antonella Forlino et al.   Nature Reviews Endocrinology

Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases

(2011) Pablo Perez-Pinera et al.   NUCLEIC ACIDS RESEARCH

Advantages of bioluminescence imaging to follow siRNA or chemotherapeutic treatments in osteosarcoma preclinical models

(2010) J Rousseau et al.   CANCER GENE THERAPY

Defining the risks of mesenchymal stromal cell therapy

(2010) Darwin J. Prockop et al.   CYTOTHERAPY

In vivo cellular adaptation to ER stress: survival strategies with double-edged consequences

(2010) K. Y. Tsang et al.   JOURNAL OF CELL SCIENCE

In utero transplantation of adult bone marrow decreases perinatal lethality and rescues the bone phenotype in the knockin murine model for classical, dominant osteogenesis imperfecta

(2009) C. Panaroni et al.   BLOOD

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Allele-specific silencing of the dominant disease allele in sialuria by RNA interference

(2008) Riko D. Klootwijk et al.   FASEB JOURNAL

Enhancement of Allele Discrimination by Introduction of Nucleotide Mismatches into siRNA in Allele-Specific Gene Silencing by RNAi

(2008) Yusuke Ohnishi et al.   PLoS One

Osteogenesis Imperfecta: Update on presentation and management

(2008) Moira S. Cheung et al.   REVIEWS IN ENDOCRINE & METABOLIC DISORDERS