Gene Therapy Gt | List of Frontiers open access articles

Original Research

Published on 28 Jan 2021

A Novel Branched DNA-Based Flowcytometric Method for Single-Cell Characterization of Gene Therapy Products and Expression of Therapeutic Genes

in Primary Immunodeficiencies

Laura Garcia-Perez
Marja C.J.A. van Eggermond
Elisa Maietta
Marie-Louise P. van der Hoorn
Karin Pike-Overzet
Frank J. T. Staal

Frontiers in Immunology

doi 10.3389/fimmu.2020.607991

10,031 views
8 citations

Original Research

Published on 09 Sep 2024

Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning

in Bone Research

Sara Penna
Alessandra Zecchillo
Martina Di Verniere
Elena Fontana
Valeria Iannello
Eleonora Palagano
Stefano Mantero
Andrea Cappelleri
Elena Rizzoli
Ludovica Santi

Frontiers in Endocrinology

doi 10.3389/fendo.2024.1450349

3,529 views
2 citations

Original Research

Published on 06 Jan 2023

National experience with adenosine deaminase deficiency related SCID in Polish children

in Primary Immunodeficiencies

Nel Dąbrowska-Leonik
Barbara Piątosa
Ewa Słomińska
Nadezda Bohynikova
Katarzyna Bernat-Sitarz
Ewa Bernatowska
Beata Wolska-Kuśnierz
Krzysztof Kałwak
Sylwia Kołtan
Anna Dąbrowska

Frontiers in Immunology

doi 10.3389/fimmu.2022.1058623

6,957 views
4 citations

Review

Published on 16 Sep 2021

Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

in Molecular Diagnostics and Therapeutics

Caroline Sevin
Kumaran Deiva

Frontiers in Molecular Biosciences

doi 10.3389/fmolb.2021.624988

10,927 views
38 citations

Review

Published on 16 Jun 2022

Gene Edited T Cell Therapies for Inborn Errors of Immunity

in Genome Editing in Blood Disorders

T. A. Fox
B. C. Houghton
C. Booth

Frontiers in Genome Editing

doi 10.3389/fgeed.2022.899294

6,066 views
7 citations

Review

Published on 09 Jan 2017

Perspective on the Road toward Gene Therapy for Parkinson’s Disease

Bas Blits
Harald Petry

Frontiers in Neuroanatomy

doi 10.3389/fnana.2016.00128

13,684 views
31 citations

Original Research

Published on 12 May 2020

Compensatory Cross-Modal Plasticity Persists After Sight Restoration

in Auditory Cognitive Neuroscience

Theresa G. Mowad
Aimee E. Willett
Mani Mahmoudian
Mikhail Lipin
Armin Heinecke
Albert M. Maguire
Jean Bennett
Manzar Ashtari

Frontiers in Neuroscience

doi 10.3389/fnins.2020.00291

10,242 views
24 citations

Original Research

Published on 21 Jul 2020

In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion

in Molecular Diagnostics and Therapeutics

Alessandra Ricca
Federica Cascino
Francesco Morena
Sabata Martino
Angela Gritti

Frontiers in Molecular Biosciences

doi 10.3389/fmolb.2020.00167

5,579 views
7 citations

Original Research

Published on 13 Nov 2023

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

in Primary Immunodeficiencies

Maria Carmina Castiello
Martina Di Verniere
Elena Draghici
Elena Fontana
Sara Penna
Lucia Sereni
Alessandra Zecchillo
Denise Minuta
Paolo Uva
Marco Zahn

Frontiers in Immunology

doi 10.3389/fimmu.2023.1268620

5,454 views
4 citations

Original Research

Published on 21 Dec 2022

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

in Children and Health

Sophie Boursange
Marco Araneda
Caroline Stalens
Isabelle Desguerre
Christine Barnerias
Marie-Christine Nougues
Arnaud Isapof
Susana Quijano-Roy
Nadia Blu Genestine
Laetitia Ouillade

Frontiers in Pediatrics

doi 10.3389/fped.2022.1062390

7,002 views
10 citations

Review

Published on 21 Jun 2022

Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

in Pediatric Neurology

Kimberly Goodspeed
Rachel M. Bailey
Suyash Prasad
Chanchal Sadhu
Jessica A. Cardenas
Mary Holmay
Deborah A. Bilder
Berge A. Minassian

Frontiers in Neurology

doi 10.3389/fneur.2022.805007

9,073 views
10 citations

Articles

A Novel Branched DNA-Based Flowcytometric Method for Single-Cell Characterization of Gene Therapy Products and Expression of Therapeutic Genes

Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning

National experience with adenosine deaminase deficiency related SCID in Polish children

Clinical Trials for Gene Therapy in Lysosomal Diseases With CNS Involvement

Gene Edited T Cell Therapies for Inborn Errors of Immunity

Perspective on the Road toward Gene Therapy for Parkinson’s Disease

Compensatory Cross-Modal Plasticity Persists After Sight Restoration

In vitro Validation of Chimeric β-Galactosylceramidase Enzymes With Improved Enzymatic Activity and Increased Secretion

Partial correction of immunodeficiency by lentiviral vector gene therapy in mouse models carrying Rag1 hypomorphic mutations

Parents' dilemma: A therapeutic decision for children with spinal muscular atrophy (SMA) type 1

Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies