Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

Lysosomal Storage Diseases: an overview

Alves, Sandra

October 2018

Lysosomal storage diseases (LSDs)are an heterogenous group rare inherited metabolic diseases caused ...

Molecular Mechanisms in Lysosomal Storage Diseases: From Pathogenesis to Therapeutic Strategies

De Pasquale, Valeria
Scarcella, Melania
Pavone, Luigi Michele

January 2022

Lysosomal storage diseases (LSDs) are a group of metabolic diseases caused by inborn mutations of ly...

Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal storage disorders (LSDs) are a group of a rare inherited metabolic disorders.N/

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Lysosomal storage diseases

Platt, F
D'Azzo, A
Davidson, B
Neufeld, E
Tifft, C

January 2018

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...

Lysosomal storage diseases

Platt, FM
D'Azzo, A
Davidson, BL
Neufeld, EF
Tifft, CJ

October 2018

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...

Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

Alves, Sandra

November 2018

Lysosomal Storage Disorders (LSDs) are a group of rare inherited metabolic diseases caused by the ma...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

Lysosomal Storage Diseases: an overview

Alves, Sandra

October 2018

Lysosomal storage diseases (LSDs)are an heterogenous group rare inherited metabolic diseases caused ...

Molecular Mechanisms in Lysosomal Storage Diseases: From Pathogenesis to Therapeutic Strategies

De Pasquale, Valeria
Scarcella, Melania
Pavone, Luigi Michele

January 2022

Lysosomal storage diseases (LSDs) are a group of metabolic diseases caused by inborn mutations of ly...

Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal storage disorders (LSDs) are a group of a rare inherited metabolic disorders.N/

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Use of an antisense-mediated exon skipping approach as a therapeutic option for a common Mucolipidosis type II causing mutation

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

September 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Development of an antisense-mediated exon skipping therapeutic strategy to correct a frequent causing mutation in Mucolipidosis II

Matos, Liliana
Vilela, Regina
Coutinho, Maria Francisca
Gaspar, Paulo
Alves, Sandra

November 2017

Lysosomal Storage Disorders (LSDs) are a group of rare inherited diseases caused by the malfunction ...

Lysosomal storage diseases

Platt, F
D'Azzo, A
Davidson, B
Neufeld, E
Tifft, C

January 2018

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...

Lysosomal storage diseases

Platt, FM
D'Azzo, A
Davidson, BL
Neufeld, EF
Tifft, CJ

October 2018

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosoma...

Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

Alves, Sandra

November 2018

Lysosomal Storage Disorders (LSDs) are a group of rare inherited metabolic diseases caused by the ma...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

New strategies for the treatment of lysosomal storage diseases

Giancarlo Parenti
Claudio Pignata
Pietro Vajro
Mariacarolina Salerno

January 2012

The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the de...

Lysosomal Storage Diseases: an overview

Alves, Sandra

October 2018

Lysosomal storage diseases (LSDs)are an heterogenous group rare inherited metabolic diseases caused ...

Molecular Mechanisms in Lysosomal Storage Diseases: From Pathogenesis to Therapeutic Strategies

De Pasquale, Valeria
Scarcella, Melania
Pavone, Luigi Michele

January 2022

Lysosomal storage diseases (LSDs) are a group of metabolic diseases caused by inborn mutations of ly...

Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC

Abstract

Extracted data

Development of an antisense-mediated exon skipping approach as a therapeutic option for the ML II-causing mutation c.3503_3504delTC

Abstract

Extracted data

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