Buss Nicholas A P S - Biologics Safety Assessment, MedImmune

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Papers共 43 篇Author StatisticsCo-AuthorSimilar Experts

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Interim Results for RGX-381: a First in Human Phase 1/2 Clinical Trial of AAV.CB7.hCLN2 Gene Therapy for CLN2 Batten Disease-Associated Retinopathy

Robert Henderson,Paul Gissen, Katrina Prise, Ayeesha Vithlani, Sarah Cornelius, Dermot Roche, Dorothy A. Thompson,Nicholas Buss, Alexander Bailey,Falabella Paulo,Wei Chieh Huang,Jenna Burke,Christina Ohnsman

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCEno. 7 (2024)

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Intrathecal or Intravenous AAV9-IDUA/RGX-111 at Minimal Effective Dose Prevents Cardiac, Skeletal and Neurologic Manifestations of Murine MPS I

Lalitha R. Belur, Avery K. Huber,Hillary Mantone, Mason Robertson,Miles C. Smith,Andrea D. Karlen,Kelley F. Kitto,Li Ou,Chester B. Whitley,Elizabeth Braunlin,Justin Furcich,Troy C. Lund,Davis Seelig,Carolyn A. Fairbanks,Nicholas Buss,Kwi Hye Kim,R. Scott Mcivor

MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENTno. 4 (2024)

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Prevention of Cardiac, Neurologic, and Skeletal Manifestations of Murine MPS I by Either Systemic or CSF-Directed AAV9-IDUA/RGX-111 Administered at Minimal Effective Dose

Molecular Therapy - Methods & Clinical Development（2024）

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RGX-381: Interim Results from the First-in-human Clinical Trial of an Investigational Gene Therapy for the Treatment of Ocular Manifestations of CLN2 Batten Disease

Christina Ohnsman, Alexander M. Bailey, Wei Huang,Nicholas Buss,Kwi Hye Kim, Gary Chan,Jenna Burke,Dawn Phillips,Mikayla Higgins,Robert H. Henderson,Paul Gissen,Angela Schulz, Stephen Pakola,Paulo Falabella

Molecular Genetics and Metabolismno. 2 (2024): 107987

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Spontaneous Seizures Associated with Cortical Interneuron Loss in Cln2R207X Mice Are Ameliorated Via Gene Therapy

Keigo Takahashi,Elizabeth M. Eultgen,Sophie H. Wang,Nicholas Rensing,Hemanth R. Nelvagal,Joshua T. Dearborn,Olivier Danos,Nicholas Buss,Mark Sands,Michael Wong,Jonathan D. Cooper

Molecular Genetics and Metabolismno. 2 (2023): 107333-107333

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Treatment of a Novel CLN2 Patient-Derived in Vitro Ocular Model with RGX-381 Gene Therapy Prevents or Reverses Lysosomal Waste Accumulation

Kwi Kim,Serena Corti,Kevin Achberger,Ryan Reddinger, Chris Tully, Wei Huang,Yevgeniya Atiskova,Stefan Liebau,Angela Schulz,Simon Dulz,Peter Loskill,Nicholas Buss,Michele Fiscella,Paulo Falabella,Christina Ohnsman

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCEno. 8 (2023)

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Characterization of Retinal Degeneration Phenotype in Classic CLN2 Disease Using OCT Biomarkers and an in Vitro Retinal Model

Christina Ohnsman,Kwi Hye Kim,Wei Chieh Huang,Serena Corti,Kevin Achberger,Stefan Liebau,Peter Loskill,Yevgeniya Atiskova,Paulo Falabella,Nicholas Buss,Angela Schulz,Simon Dulz

MOLECULAR GENETICS AND METABOLISM（2023）

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