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Open access

Research article

First published 2013

Knowledge Discovery in Variant Databases Using Inductive Logic Programming

Hoan Nguyen, Tien-Dao Luu, Olivier Poch [...]

Bioinformatics and Biology Insights

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Research article

First published 2015-08

Generation of Human Induced Pluripotent Stem Cells from Extraembryonic Tissues of Fetuses Affected by Monogenic Diseases

Paola Spitalieri, Rosa V. Talarico, Annalisa Botta [...]

Cellular Reprogramming

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Research article

First published 2010-08

Chromosomal Distribution of Disease Genes in the Human Genome

David N. Cooper, Edward V. Ball, Matthew Mort

Genetic Testing and Molecular Biomarkers

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Review article

First published 2022-10

Disease-Targeted Treatment Translation in Fragile X Syndrome as a Model for Neurodevelopmental Disorders

Elizabeth Berry-Kravis

Journal of Child Neurology

Retricted access

Review article

First published 2014-07

Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy

Pádraig J. Mulcahy, Kayleigh Iremonger, Evangelia Karyka [...]

Human Gene Therapy

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Research article

First published 2000-11

Genetic Correction of Inherited Epidermal Disorders

Paul A. Khavari

Human Gene Therapy

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Research article

First published 2020-08

Gene Editing for Treatment and Prevention of Human Diseases: A Global Survey of Gene Editing-Related Researchers

Leonardo Fernandes Moutinho Rocha, Luiza Amara Maciel Braga, Fabio Batista Mota

Human Gene Therapy

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Review article

First published 2015-03

A Review of Gene Therapy in Canine and Feline Models of Lysosomal Storage Disorders

Allison M. Bradbury, Brittney L. Gurda, Margret L. Casal [...]

Human Gene Therapy Clinical Development

Free access

Research article

First published 2018-12

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

Basel T. Assaf, Laurence O. Whiteley

Toxicologic Pathology

Free access

Review article

First published 2007-10

Review: Genetics of the cardiometabolic syndrome: new insights and therapeutic implications

Silvia Sookoian, Carlos J. Pirola

Therapeutic Advances in Cardiovascular Disease

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