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Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 30, 2024
Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression
Ignazio Fiduccia, Federica Corrao, Maria Grazia Zizzo, et al.
International Journal of Molecular Sciences
|
October 28, 2023
Readthrough Approach Using NV Translational Readthrough-Inducing Drugs (TRIDs): A Study of the Possible Off-Target Effects on Natural Termination Codons (NTCs) on TP53 and Housekeeping Gene Expression
Riccardo Perriera, Emanuele Vitale, Ivana Pibiri, et al.
International Journal of Molecular Sciences
|
June 10, 2023
Investigating the Inhibition of FTSJ1, a Tryptophan tRNA-Specific 2'-O-Methyltransferase by NV TRIDs, as a Mechanism of Readthrough in Nonsense Mutated CFTR
Pietro Salvatore Carollo, Marco Tutone, Giulia Culletta, et al.
Molecular Therapy. Nucleic Acids
|
January 16, 2026
Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency
Ignazio Fiduccia, Emanuele Vitale, Riccardo Varrica, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 30, 2025
A precision medicine approach to primary immunodeficiency disease: Ataluren strikes nonsense mutations once again
Laura Lentini, Riccardo Perriera, Federica Corrao, et al.
Biomedicines
|
April 23, 2022
Novel Translational Read-through-Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome
Valentino Bezzerri, Laura Lentini, Martina Api, et al.
Biomedicine & Pharmacotherapy = Biomedecine & Pharmacotherapie
|
October 20, 2022
Nonsense codons suppression. An acute toxicity study of three optimized TRIDs in murine model, safety and tolerability evaluation
Federica Corrao, Maria Grazia Zizzo, Marco Tutone, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
November 16, 2025
Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues
Valeria Rimoldi, Giulia Soldà, Anita Capalbo, et al.
Journal of Child Neurology
|
October 21, 2024
Stress and Quality of Life of Parents of Children With POLR3-Related Leukodystrophy: A Cross-Sectional Pilot Study
Laura Lentini, Helia Toutounchi, Alexandra Chapleau, et al.
Neurology. Genetics
|
October 27, 2025
Developing a National Network for Leukodystrophy Research and Care in Canada: The CARELeuko Initiative
Alexandra Chapleau, Adam Le, Justin Simo, et al.
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of 5
Search research articles
Search
Showing results (41-50 of 50) with videos related to
Sort By:
Page
of 5
You have reached the last page of results.
This site can display upto 50 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 30, 2024
Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression
Ignazio Fiduccia, Federica Corrao, Maria Grazia Zizzo, et al.
International Journal of Molecular Sciences
|
October 28, 2023
Readthrough Approach Using NV Translational Readthrough-Inducing Drugs (TRIDs): A Study of the Possible Off-Target Effects on Natural Termination Codons (NTCs) on TP53 and Housekeeping Gene Expression
Riccardo Perriera, Emanuele Vitale, Ivana Pibiri, et al.
International Journal of Molecular Sciences
|
June 10, 2023
Investigating the Inhibition of FTSJ1, a Tryptophan tRNA-Specific 2'-O-Methyltransferase by NV TRIDs, as a Mechanism of Readthrough in Nonsense Mutated CFTR
Pietro Salvatore Carollo, Marco Tutone, Giulia Culletta, et al.
Molecular Therapy. Nucleic Acids
|
January 16, 2026
Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency
Ignazio Fiduccia, Emanuele Vitale, Riccardo Varrica, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 30, 2025
A precision medicine approach to primary immunodeficiency disease: Ataluren strikes nonsense mutations once again
Laura Lentini, Riccardo Perriera, Federica Corrao, et al.
Biomedicines
|
April 23, 2022
Novel Translational Read-through-Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome
Valentino Bezzerri, Laura Lentini, Martina Api, et al.
Biomedicine & Pharmacotherapy = Biomedecine & Pharmacotherapie
|
October 20, 2022
Nonsense codons suppression. An acute toxicity study of three optimized TRIDs in murine model, safety and tolerability evaluation
Federica Corrao, Maria Grazia Zizzo, Marco Tutone, et al.
Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|
November 16, 2025
Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues
Valeria Rimoldi, Giulia Soldà, Anita Capalbo, et al.
Journal of Child Neurology
|
October 21, 2024
Stress and Quality of Life of Parents of Children With POLR3-Related Leukodystrophy: A Cross-Sectional Pilot Study
Laura Lentini, Helia Toutounchi, Alexandra Chapleau, et al.
Neurology. Genetics
|
October 27, 2025
Developing a National Network for Leukodystrophy Research and Care in Canada: The CARELeuko Initiative
Alexandra Chapleau, Adam Le, Justin Simo, et al.
Page
of 5