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 […]

Successful delivery of large-size CRISPR/Cas9 vectors in hard-to-transfect human cells using small plasmids

Successful delivery of large-size CRISPR/Cas9 vectors in hard-to-transfect human cells using small plasmids

With the rise of recent highly effective genome engineering applied sciences, equivalent to CRISPR/Cas9, cell fashions might be engineered successfully to speed up fundamental and illness analysis. Essentially the most essential step on this process is the environment friendly supply of international nucleic acids into cells by mobile transfection. For the reason that vectors encoding […]

Production of lentiviral vectors in suspension cells using low proportion of supercoiled circular plasmid DNA

Production of lentiviral vectors in suspension cells using low proportion of supercoiled circular plasmid DNA

The supercoiled round (SC) topology type of plasmid DNA has been regarded to be advantageous over open round or linearized analogue in transfection and expression effectivity, and due to this fact are largely demanded within the biopharmaceutical manufacturing. Nevertheless, manufacturing of high-purity SC plasmid DNA would lead to excessive manufacturing value. The impact of SC […]

Preclinical safety evaluation of a recombinant plasmid vector encoding mature human neutrophil peptide-1 by repeated local administrations in nonhuman primates

Preclinical safety evaluation of a recombinant plasmid vector encoding mature human neutrophil peptide-1 by repeated local administrations in nonhuman primates

In our earlier research, a novel gene remedy method was developed based mostly on a plasmid vector pSecTag2B through which recombinant HNP1 gene was regulated below a cytomegalovirus promoter to encode a mature HNP1 type. We confirmed for the primary time in numerous tumor fashions together with human most cancers xenografts that overexpression of HNP1 […]