A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

Mutations within the fukutin-related protein (FKRP) gene end in a broad spectrum of muscular dystrophy (MD) phenotypes, together with the extreme Walker-Warburg syndrome (WWS). Right here, we develop a gene-editing method that replaces your complete mutant open studying body with the wild-type sequence to universally right all FKRP mutations. We apply this method to right […]

Exploiting Single-Cell Tools in Gene and Cell Therapy

Exploiting Single-Cell Tools in Gene and Cell Therapy

Single-cell molecular instruments have been developed at an unimaginable tempo over the past 5 years as sequencing prices proceed to drop and quite a few molecular assays have been coupled to sequencing readouts. This fast interval of technological growth has facilitated the delineation of particular person molecular traits together with the genome, transcriptome, epigenome, and […]

Review of the Molecular Genetics of Basal Cell Carcinoma; Inherited Susceptibility, Somatic Mutations, and Targeted Therapeutics

Review of the Molecular Genetics of Basal Cell Carcinoma; Inherited Susceptibility, Somatic Mutations, and Targeted Therapeutics

Basal cell carcinoma (BCC) is a major public well being concern, with greater than three million circumstances occurring every year in america, and with an growing incidence. The molecular foundation of BCC is advanced, involving an interaction of inherited genetic susceptibility, together with single nucleotide polymorphisms and genetic syndromes, and sporadic somatic mutations, typically induced […]

Comprehensive Study Identifies a Sensitive, Low-Risk, Closed-System Model for Detection of Fungal Contaminants in Cell and Gene Therapy Products

Comprehensive Study Identifies a Sensitive, Low-Risk, Closed-System Model for Detection of Fungal Contaminants in Cell and Gene Therapy Products

The US Meals and Drug Administration (FDA) regulates manufacturing and testing of superior therapeutic medicinal merchandise (ATMPs) to make sure the security of every product for human use. Gold normal sterility testing (USP<71>) and different blood tradition methods have main limitations for the detection of fungal contaminants. On this research, we evaluated the efficiency of iLYM […]

Short regulatory DNA sequences to target brain endothelial cells for gene therapy

Short regulatory DNA sequences to target brain endothelial cells for gene therapy

Gene vectors focusing on CNS endothelial cells enable to govern the blood-brain barrier and to right genetic defects within the CNS. As a result of vectors based mostly on the adeno-associated virus (AAV) have a restricted capability, it’s important that the DNA sequence controlling gene expression is brief. As well as, it should be particular […]

Investigating the barrier activity of novel, human enhancer-blocking chromatin insulators for hematopoietic stem cell gene therapy

Anti Human IREM 2

Clone: ​​UP-H2 Isotype: IgG1 Tested Applications: FCM Conjugate: APC Immunogen: cells transfected with IREM-2-HA Species reactivity: human Storage buffer: the reagent is provided in an aqueous buffered solution containing protein stabilizer and sodium azide (NaN3) ≤0.09% Regulatory status: IVD-CE Molecular weight: 34 KDa Product-specific information Description: The monoclonal antibody UP-H2 recognizes human IREM-2 (immune receptor […]

Short regulatory DNA sequences to target brain endothelial cells for gene therapy

Exosome-Depleted FBS Media Supplement

Abstract Extracellular vesicles (EVs), including nano-sized exosomes, have the ability to transfer multiple functional molecules between cells. In cell culture experiments, fetal bovine serum (FBS) is often used to supplement the cell culture medium as a nutrient, but it is important to know that FBS also contains significant amounts of EV. The objective of the […]

Comprehensive Study Identifies a Sensitive, Low-Risk, Closed-System Model for Detection of Fungal Contaminants in Cell and Gene Therapy Products

Sheep Anti-Mullerian Hormone, AMH BT-LAB Kit

Product name Anti-Mullerian Hormone (AMH), ELISA Kit Full Product Name Sheep Anti-Müllerian Hormone (AMH) ELISA Kit Product gene name AMH ELISA kit Species reactivity: Sheep Samples Serum, plasma, cell culture supernatants, cell lysates, tissue homogenates Test type: Quantitative sandwich Detection range: 0.05 ng / ml – 15 ng / ml Sensitivity: 0.02 ng / ml […]

Review of the Molecular Genetics of Basal Cell Carcinoma; Inherited Susceptibility, Somatic Mutations, and Targeted Therapeutics

EpiQuik Circulating Total Histone H3 Quantification Kit (Colorimetric)

The EpiQuik Circulating Total Histone H3 Quantitation Kit (Colorimetric) is a complete set of optimized buffers and reagents specifically designed to quantify the levels of histone H3 proteins regardless of their modified state and can also be used to normalize the content of modified histone H3 of samples when run in parallel with the Epigentek […]