Category Archives: news

Detail Introduction: CRISPR-Cas9 system is a widely used tool to perform genetic editing. In recent years, this technology has been well developed. Specifically, deactivated nuclease-null Cas9 (dCas9) together with guide RNA (gRNA) can be utilized to convey various transcriptional or epigenetic modulators to the target site to regulate gene expression. One of these modulators is […]

Detail Introduction: Retinitis pigmentosa (RP) characterized as retina degeneration is one of the major causes for inherited blindness, which can occur in one of four thousand babies. Most of RP show rod photoreceptor dysfunctions induced by gene mutations, which can further trigger loss of cone photoreceptors and then result in blindness. To date, there is […]

Detail Introduction: Recombinant adeno-associated virus (AAV) vectors exhibit promising outcomes on gene therapy with its durable expression in the targeting tissues. Even so, in most cases, AAV immunogenicity still represents a major limitation for its clinical trials. Recently, a research team from Sorbonne Université and INSERM U974 (France) has employed an effective means to avoid […]

Detail Introduction:Retroviral replicating vectors (RRVs) has been proved as an excellent gene therapy vector to achieve efficient gene transduction. To date, more than 499 clinical trials have been carried out using lentivirus vectors for gene delivery and promising gene therapy outcomes have been achieved in a variety of diseases, mostly in cancer. Today, a dual-vector […]

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, […]

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 […]

Detail Introduction: Duchenne muscular dystrophy (DMD), caused by out-of-frame mutations of dystrophin gene, is a progressive degenerative disease resulting in cardiac and smooth muscle dysfunction in approximately 250–300,000 boys and young men worldwide. Previous studies showed that AAV-delivered CRISPR gene editing tools can reframe the mutated dystrophin gene and restore dystrophin expression in vitro and […]

Detail Introduction: Around one in 1000 newborns are suffered from congenital disabling hearing loss, among which, mutations in OTOF gene (encoding otoferlin protein) accounting for 2.3–10% of patients, and over one thousand pathogenic mutations have been identified within this gene. Therefore, a postnatal supplement of otoferlin cDNA into the inner ear might be a promising […]

Detail Introduction: Duchenne muscular dystrophy (DMD), owing to the gene mutation of dystrophin, is a severe genetic disease resulting in body-wide muscle degeneration and necrosis in boys and young men. Lots of evidence demonstrates that an important early pathogenic event from DMD patients is the abnormal elevation of intracellular Ca2+, resulting from the dysfunction of […]