Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce, +29

New England Journal of Medicine

Ocular DiseaseGeneticsImmunologyPathologyDisease Gene Identification +16

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Albert M. Maguire, Katherine A. High, Alberto Auricchio, +30

The Lancet

Gene TherapiesGenetic DisorderGeneticsClinical TrialsTranslational Research +5

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Francesca Simonelli, Albert M. Maguire, Francesco Testa, +19

Molecular Therapy

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Jean Bennett, Jennifer Wellman, Kathleen Marshall, +19

The Lancet

Translational MedicineGene TherapiesOcular DiseaseAav2 Gene TherapyIn Vivo Gene Therapy +5

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Jean Bennett, Manzar Ashtari, Jennifer Wellman, +17

Science Translational Medicine

Gene TherapiesTranslational MedicineRpe65 GeneRetinaIn Vivo Gene Therapy +11

Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2

Francesco Testa, Albert M. Maguire, Settimio Rossi, +13

Ophthalmology

Adeno-associated VirusThree-year Follow-upNeurovirologyPathologyVirology +2

Type 1 and type 2 cytokine regulation of macrophage endocytosis: differential activation by IL-4/IL-13 as opposed to IFN-gamma or IL-10.

Luis J. Montaner, R. P. Da Silva, Junwei Sun, +4

PubMed

Macrophage EndocytosisImmunologyImmune RegulationImmunologic MechanismCytoskeleton +20

Gene gun delivery of mRNA in situ results in efficient transgene expression and genetic immunization.

Ping Qiu, Pamela R. Ziegelhoffer, Junwei Sun, +1

PubMed

Type 1 and Type 2 Cytokine Regulation of Macrophage Endocytosis: Differential Activation by IL-4/IL-13 as Opposed to IFN-γ or IL-10

Luis J. Montaner, Rosângela P. da Silva, Junwei Sun, +4

The Journal of Immunology

Immune RegulationImmunologyImmunologic MechanismCytoskeletonInnate Immunity +23

Enhancement of Human Immunodeficiency Virus Type 1–Specific CD4 and CD8 T Cell Responses in Chronically Infected Persons after Temporary Treatment Interruption

Emmanouil Papasavvas, Gabriel M. Ortiz, Robert Gross, +11

The Journal of Infectious Diseases