Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

Sandra Duqué, Béatrice Joussemet, Christel Rivière, +7

Molecular Therapy

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Elisa Dominguez, Thibaut Marais, Nicolas Chatauret, +8

Human Molecular Genetics

Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression <i>in vitro</i> and <i>in vivo</i>

Stéphanie Philippe, Chamsy Sarkis, Martine Barkats, +5

Proceedings of the National Academy of Sciences

Viral ReplicationSystems BiologyDefective IntegraseNeurovirologyMedicine +13

Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology

Inger Lauritzen, Raphaëlle Pardossi‐Piquard, Alexandre Bourgeois, +7

Acta Neuropathologica

Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases

Nadine Dragin, Jacky Bismuth, Géraldine Cizeron-Clairac, +9

Journal of Clinical Investigation

Human ThymusLymphocyte DevelopmentImmunologyImmune RegulationFemale Reproductive Function +25

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase

Francesco Puzzo, Pasqualina Colella, Maria Grazia Biferi, +22

Science Translational Medicine

Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice

Sofia Benkhelifa‐Ziyyat, Aurore Besse, Marianne Roda, +5

Molecular Therapy

Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice

Yannick Tanguy, Maria Grazia Biferi, Aurore Besse, +4

Frontiers in Molecular Neuroscience

Systemic Aavrh10ImmunologyTransduction EfficiencyCns TransductionSocial Sciences +14

The Nuclear Factor κB–Activator Gene PLEKHG5 Is Mutated in a Form of Autosomal Recessive Lower Motor Neuron Disease with Childhood Onset

Isabelle Maystadt, René Rezsöhazy, Martine Barkats, +9

The American Journal of Human Genetics

Neurodegenerative DiseasesNeurobiology Of DiseaseGenetic DisorderGeneticsMotor Development +8

Interferon beta induces clearance of mutant ataxin 7 and improves locomotion in SCA7 knock-in mice

Alice Chort, Sandro Alves, Martina Marinello, +12

Brain

Pml Protein ExpressionMolecular RegulationImmunologyCell DeathCellular Physiology +21