Advertisement

Topics

Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis

2019-05-21 20:22:18 | BioPortfolio

Published on BioPortfolio: 2019-05-21T20:22:18-0400

Clinical Trials [149 Associated Clinical Trials listed on BioPortfolio]

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

The purpose of this study is to determine if it is safe to administer unrelated umbilical cord blood to pregnant women in their first trimester of pregnancy with a fetus that has a known d...

Gene Transfer Study of AAV9-CLN3 for Treatment NCL Type 3

Open-label, single dose, dose-escalation clinical trial AAV9-CLN3 via intrathecal injection in NCL type 3 subjects

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Aim is to undertake a screening study that identifies undiagnosed patients with LSDs and determine the prevalence of these diseases with special focus on underrepresented minority groups.

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

The purpose of this study is to evaluate the effect of small molecule therapy in primary cells derived from patients with lysosomal storage disease. The study will focus on activity of sma...

Gene Therapy for Male Patients With Danon Disease Using RP-A501; AAV9.LAMP2B

This is a non-randomized open-label Phase 1 study to evaluate the safety and toxicity of gene therapy using a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lyso...

PubMed Articles [8143 Associated PubMed Articles listed on BioPortfolio]

Lysosomal diseases: Overview on current diagnosis and treatment.

Lysosomal diseases (LDs), also known as lysosomal storage diseases (LSDs), are a heterogeneous group of conditions caused by defects in lysosomal function. LDs may result from deficiency of lysosomal ...

Transparenchymal renal pelvis injection of recombinant AAV9 vectors is a practical approach for gene delivery in the kidney.

Gene therapy has great potential in treating human diseases, but little progress has been made in preclinical and clinical studies of renal diseases. To find an effective gene delivery approach in the...

Lysosomal storage disorders affecting the heart: a review.

Lysosomal storage disorders (LSD) comprise a group of diseases caused by a deficiency of lysosomal enzymes, membrane transporters or other proteins involved in lysosomal biology. Lysosomal storage dis...

Mitochondrial biogenesis is transcriptionally repressed in lysosomal lipid storage diseases.

Perturbations in mitochondrial function and homeostasis are pervasive in lysosomal storage diseases, but the underlying mechanisms remain unknown. Here, we report a transcriptional program that repres...

TRPML1-/TFEB-Dependent Regulation of Lysosomal Exocytosis.

Emerging experimental evidences indicate that the lysosome can trigger a calcium signaling, via TRPML1/calcineurin/TFEB pathway, that promotes lysosomal exocytosis and clearance of lysosomal accumulat...

Medical and Biotech [MESH] Definitions

Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.

An abundant lysosomal-associated membrane protein that has been found to shuttle between LYSOSOMES; ENDOSOMES; and the PLASMA MEMBRANE. Loss of expression of lysosomal-associated membrane protein 2 is associated with GLYCOGEN STORAGE DISEASE TYPE IIB.

Group of lysosomal storage diseases each caused by an inherited deficiency of an enzyme involved in the degradation of glycosaminoglycans (mucopolysaccharides). The diseases are progressive and often display a wide spectrum of clinical severity within one enzyme deficiency.

A lysosomal-associated membrane glycoprotein that is expressed at high levels in mature DENDRITIC CELLS.

An abundant lysosomal-associated membrane protein that has been found to shuttle between LYSOSOMES; ENDOSOMES; and the PLASMA MEMBRANE. In PLATELETS and T-LYMPHOCYTES it may play a role in the cellular degranulation process.

More From BioPortfolio on "Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis"

Advertisement
Quick Search
Advertisement
Advertisement

 

Searches Linking to this Trial