Author Archives: shanzhu

What is Adeno-associated virus (AAV) vector?

AAV protocol Download Adeno Associated Virus (AAV) Adeno-associated virus (AAV) is a small-single strand DNA virus, member of human parvovirus, originally described in the 1960s by Bob Atchison at Pittsburgh and Wallace Rowe at NIH as a contaminant in preparation of adenovirus [1,2]. Shortly after its discovery, AAV was then isolated from humans, but serological studies […]

Internal Gene Editing Field Analysis

CRISPR/Cas9 premade products CRISPR Editas Medicines Genome editing firm that researches and develops therapies for treating genetic based diseases primarily using CRISPR technology.  CEO: Cynthia Collins  Website: https://www.editasmedicine.com/  Located: Cambridge, Massachusetts   Employees: 133  Market Cap: $22.06 (use link for updated value)  Annual Revenue (2018): $30.1M  Funding History: Series A funding of $43M via Flagship Ventures achieved in November 2013, Series B funding of $120M […]

Transfection Protocol

Protocol Download Introduction to LipoGene Transfectin Reagent GeneMedi provide LipoGeneTM transfection reagent which is highly potent cationic lipofection reagent that has been shown to effectively transfect plasmids or siRNA, as well as nucleic acid-protein complexes, into cultured adherent and suspension cell lines. Researchers use LipoGeneTM Reagent for siRNA- and shRNA-based gene knockdown experiments, as well as […]

Atrial-Specific Gene Delivery Using an AAV Vector

Detail Introduction: Atrial diseases pose a major threaten to human health. Recently Ni L et al established an atrial-specific genetic manipulation system based on AAV (adeno-associated virus) vectors, which facilitates the pathogenesis study of atrial diseases and provides a powerful genetic tool for gene therapy study. The correlated findings were published in the journal of […]

Deamidation of Amino Acids on the Surface of AAV Capsids Leads to Charge Heterogeneity and Altered Vector Function

Detail Introduction: As an excellent gene therapy vector, AAV has been widely used for the treatment of many diseases, such as retinal dystrophy, cystic fibrosis, Duchenne Muscular Dystrophy, Hemophilia. However, post-translational modification of the AAV capsids and how they affect vector activity is poorly understood during the development of AAV vectors into pharmaceutical products. Recently, […]

Correction of half the cardiomyocytes fully rescue Friedreich Ataxia mitochondrial cardiomyopathy

Detail Introduction: Friedreich ataxia (FA) is a rare, neurodegenerative, autosomal recessive disease, characterized by spinocerebellar and sensory ataxia, including diabetes and cardiac hypertrophy, which is a major medical concern in FA, responsible for 59% of premature death of patients. FA is mainly caused by a (GAA)n expansion within the 1st intron of the frataxin gene […]