Samia R. Martin

Evry, 91002, France

Last Updated: 09/26/2026

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Found 20 publications

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome.

Journal: Molecular therapy. Methods & clinical development
Published: October 09, 2018

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.

Journal: Molecular therapy. Methods & clinical development
Published: August 09, 2018

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.

An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype.

Journal: Molecular therapy. Methods & clinical development
Published: June 26, 2018

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice.

Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice.

Journal: Journal of neuropathology and experimental neurology
Published: February 07, 2018

The TGFβ pathway is a key player for the endothelial-to-hematopoietic transition in the embryonic aorta.

The TGFβ pathway is a key player for the endothelial-to-hematopoietic transition in the embryonic aorta.

Journal: Developmental biology
Published: October 11, 2017

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

Journal: Blood advances
Published: September 01, 2017

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates.

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates.

Journal: Translational research : the journal of laboratory and clinical medicine
Published: January 19, 2017

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

Journal: Molecular therapy. Nucleic acids
Published: November 12, 2016

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Journal: Nature communications
Published: August 18, 2016

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Journal: Molecular therapy. Methods & clinical development
Published: May 22, 2016

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Journal: Molecular therapy. Methods & clinical development
Published: April 26, 2016

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Journal: Science translational medicine
Published: March 06, 2015
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Last Updated: 09/26/2026