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Biomedicines
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October 28, 2023
MicroRNA Profile, Putative Diagnostic Biomarkers and RNA-Based Therapies in the Inherited Lipid Storage Disease Niemann-Pick Type C
Marisa Encarnação, Hugo David, Maria Francisca Coutinho, et al.
International Journal of Molecular Sciences
|
October 14, 2023
Development of Engineered-U1 snRNA Therapies: Current Status
Mariana Gonçalves, Juliana Inês Santos, Maria Francisca Coutinho, et al.
Genes
|
November 25, 2023
Challenges in the Definitive Diagnosis of Niemann-Pick Type C-Leaky Variants and Alternative Transcripts
Marisa Encarnação, Isaura Ribeiro, Hugo David, et al.
World Journal of Pediatrics : WJP
|
January 20, 2017
Update of the spectrum of mucopolysaccharidoses type III in Tunisia: identification of three novel mutations and in silico structural analysis of the missense mutations
Souad Ouesleti, Maria Francisca Coutinho, Isaura Ribeiro, et al.
Journal of Pediatric Endocrinology & Metabolism : JPEM
|
October 7, 2016
Solving a case of allelic dropout in the GNPTAB gene: implications in the molecular diagnosis of mucolipidosis type III alpha/beta
Maria Francisca Coutinho, Marisa Encarnação, Francisco Laranjeira, et al.
Gene
|
April 2, 2014
Prenatal skeletal dysplasia phenotype in severe MLII alpha/beta with novel GNPTAB mutation
Shagun Aggarwal, Maria Francisca Coutinho, Ashwin B Dalal, et al.
Life (Basel, Switzerland)
|
May 28, 2022
Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses Example
Juliana Inês Santos, Mariana Gonçalves, Liliana Matos, et al.
Human Gene Therapy
|
April 15, 2020
Development of an Antisense Oligonucleotide-Mediated Exon Skipping Therapeutic Strategy for Mucolipidosis II: Validation at RNA Level
Liliana Matos, Regina Vilela, Melissa Rocha, et al.
Indian Journal of Pediatrics
|
October 28, 2016
I Cell Disease (Mucolipidosis II Alpha/Beta): From Screening to Molecular Diagnosis
Ankur Singh, Rajniti Prasad, Aditya Kumar Gupta, et al.
Human Mutation
|
December 31, 2013
Mucolipidosis II-related mutations inhibit the exit from the endoplasmic reticulum and proteolytic cleavage of GlcNAc-1-phosphotransferase precursor protein (GNPTAB)
Raffaella De Pace, Maria Francisca Coutinho, Friedrich Koch-Nolte, et al.
Page
of 4
Search research articles
Search
Showing results (11-20 of 33) with videos related to
Sort By:
Page
of 4
Biomedicines
|
October 28, 2023
MicroRNA Profile, Putative Diagnostic Biomarkers and RNA-Based Therapies in the Inherited Lipid Storage Disease Niemann-Pick Type C
Marisa Encarnação, Hugo David, Maria Francisca Coutinho, et al.
International Journal of Molecular Sciences
|
October 14, 2023
Development of Engineered-U1 snRNA Therapies: Current Status
Mariana Gonçalves, Juliana Inês Santos, Maria Francisca Coutinho, et al.
Genes
|
November 25, 2023
Challenges in the Definitive Diagnosis of Niemann-Pick Type C-Leaky Variants and Alternative Transcripts
Marisa Encarnação, Isaura Ribeiro, Hugo David, et al.
World Journal of Pediatrics : WJP
|
January 20, 2017
Update of the spectrum of mucopolysaccharidoses type III in Tunisia: identification of three novel mutations and in silico structural analysis of the missense mutations
Souad Ouesleti, Maria Francisca Coutinho, Isaura Ribeiro, et al.
Journal of Pediatric Endocrinology & Metabolism : JPEM
|
October 7, 2016
Solving a case of allelic dropout in the GNPTAB gene: implications in the molecular diagnosis of mucolipidosis type III alpha/beta
Maria Francisca Coutinho, Marisa Encarnação, Francisco Laranjeira, et al.
Gene
|
April 2, 2014
Prenatal skeletal dysplasia phenotype in severe MLII alpha/beta with novel GNPTAB mutation
Shagun Aggarwal, Maria Francisca Coutinho, Ashwin B Dalal, et al.
Life (Basel, Switzerland)
|
May 28, 2022
Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses Example
Juliana Inês Santos, Mariana Gonçalves, Liliana Matos, et al.
Human Gene Therapy
|
April 15, 2020
Development of an Antisense Oligonucleotide-Mediated Exon Skipping Therapeutic Strategy for Mucolipidosis II: Validation at RNA Level
Liliana Matos, Regina Vilela, Melissa Rocha, et al.
Indian Journal of Pediatrics
|
October 28, 2016
I Cell Disease (Mucolipidosis II Alpha/Beta): From Screening to Molecular Diagnosis
Ankur Singh, Rajniti Prasad, Aditya Kumar Gupta, et al.
Human Mutation
|
December 31, 2013
Mucolipidosis II-related mutations inhibit the exit from the endoplasmic reticulum and proteolytic cleavage of GlcNAc-1-phosphotransferase precursor protein (GNPTAB)
Raffaella De Pace, Maria Francisca Coutinho, Friedrich Koch-Nolte, et al.
Page
of 4