Sage Journals: Discover world-class research

Open access

Research article

First published 2020-12

Gene Replacement Therapy: A Primer for the Health-system Pharmacist

John Petrich, Dominic Marchese, Chris Jenkins [...]

Journal of Pharmacy Practice

Retricted access

Research article

First published 2004-09

Adenoviral Vectors for Gene Replacement Therapy

Huibi Cao, David R. Koehler, Jim Hu

Viral Immunology

Retricted access

Other

First published 2016-03

Dystrophin Gene Replacement and Gene Repair Therapy for Duchenne Muscular Dystrophy in 2016: An Interview

Dongsheng Duan

Human Gene Therapy Clinical Development

Open access

Research article

First published 2024-01-02

Parental Experiences with Newborn Screening and Gene Replacement Therapy for Spinal Muscular Atrophy

Alayne P. Meyer, Anne M. Connolly, Kathryn Vannatta [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2010-08

Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in a Murine Model of Leber Congenital Amaurosis

Basil S. Pawlyk, Oleg V. Bulgakov, Xiaoqing Liu [...]

Human Gene Therapy

Retricted access

Research article

First published 2011-01

Effect of Estrogen Receptor β A1730G Polymorphism on ABCA1 Gene Expression Response to Postmenopausal Hormone Replacement Therapy

Maryam Darabi, Mohsen Ani, Mojtaba Panjehpour [...]

Genetic Testing and Molecular Biomarkers

Retricted access

Other

First published 2001-9

Murine Mucopolysaccharidosis VII: Impact of Therapies on the Phenotype,Clinical Course,and Pathology in a Model of a Lysosomal Storage Disease

Carole Vogler, Jane Barker, Mark S. Sands [...]

Pediatric and Developmental Pathology

Free access

Review article

First published 2010-10

Review: Therapeutic approaches for lysosomal storage diseases

Gregory M. Pastores

Therapeutic Advances in Endocrinology and Metabolism

Free access

Research article

First published 2010-5

The influence of the genetic variant within miRNA-binding site in estrogen receptor alpha gene on the risk of breast cancer in postmenopausal women on hormone replacement therapy

Jasmina-Ziva Cerne, Ksenija Gersak, Srdjan Novakovic

Cancer Biomarkers

Retricted access

Review article

First published 2025

Latest Advancements in Treatment Options for Infantile-Onset Pompe Disease: A Comprehensive Narrative Review

Misha Khan, Mufliha Ibrahim, Farhana Riaz [...]

Clinical Medicine Insights: Pediatrics

Search results