Particularities of lysosomal storage diseases gene therapy

Widespread distribution of beta-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol.

Gene therapy·2003

Endothelial nitric oxide synthase gene polymorphisms in Fabry's disease.

Clinical genetics·2002

Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy.

Human molecular genetics·2002

From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system.

Annals of medicine·2001

Gene therapy in lysosomal diseases.

Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie·2000

Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlation.

Neurology·2000

Mapping the global trends and hotspots of research on gene therapy and hair cell regeneration for hearing loss: a comprehensive data-mining-based study.

Gene therapy·2026

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

Gene therapy·2026

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Gene therapy·2026

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Gene therapy·2026

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

Gene therapy·2026

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

Gene therapy·2026

[Development of universal off-the-shelf T cell therapies derived from ES/iPS cells for leukemia and COVID-19].

[Rinsho ketsueki] The Japanese journal of clinical hematology·2026

[Intrathecal immunoglobulin administration for refractory viral central nervous system infection after allogeneic hematopoietic stem cell transplantation].

[Rinsho ketsueki] The Japanese journal of clinical hematology·2026

Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis With Cardiomyopathy: Insights From HELIOS-B.

Journal of the American College of Cardiology·2026

Gene Silencer Therapy in Transthyretin Amyloid Cardiomyopathy: A Meta-Analysis of Outcomes Trials.

JAMA·2026

Advanced therapy medicinal products in Saudi Arabia: Regulation and development.

Regenerative therapy·2026

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands.

Neuromuscular disorders : NMD·2026

Related Experiment Videos

Particularities of lysosomal storage diseases gene therapy

Related Concept Videos

Widespread distribution of beta-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol.

Endothelial nitric oxide synthase gene polymorphisms in Fabry's disease.

Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy.

From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system.

Gene therapy in lysosomal diseases.

Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlation.

Mapping the global trends and hotspots of research on gene therapy and hair cell regeneration for hearing loss: a comprehensive data-mining-based study.

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

[Development of universal off-the-shelf T cell therapies derived from ES/iPS cells for leukemia and COVID-19].

[Intrathecal immunoglobulin administration for refractory viral central nervous system infection after allogeneic hematopoietic stem cell transplantation].

Effect of Vutrisiran According to Baseline Tafamidis Use in Transthyretin Amyloidosis With Cardiomyopathy: Insights From HELIOS-B.

Gene Silencer Therapy in Transthyretin Amyloid Cardiomyopathy: A Meta-Analysis of Outcomes Trials.

Advanced therapy medicinal products in Saudi Arabia: Regulation and development.

289th ENMC international workshop: assessing and managing emerging AAV related toxicities after gene therapy for neuromuscular disorders, 26 - 28 September 2025, Hoofddorp, The Netherlands.

Related Experiment Videos

Particularities of lysosomal storage diseases gene therapy

Related Concept Videos

Related Articles