• Adenovirus Service • AAV Service • Lentivirus Service • Retrovirus Service
Recently, the team led by Professor Yilai Shu from the Affiliated Eye, Ear, Nose and Throat Hospital of Fudan University in China published a research paper titled "A base editor for the long-term restoration of auditory function in mice with recessive profound deafness" in the journal Nature Biomedical Engineering. The study used adenine base editor (ABE)-mediated gene editing therapy to effectively repair the Otof pathogenic mutation in the deaf mouse model and restore the expression level of otoferlin in 88% of the inner hair cells of the inner ear. At the same time, it improved the synaptic exocytosis function of the inner hair cells of the inner ear and restored hearing to a level close to the wild type for up to 1.5 years without obvious off-target effects. It is reported that this is the longest-observed effective result in the field of gene therapy for deafness in animal models to date.
Although CAR-T cell therapy has brought revolutionary progress to the field of cancer treatment, especially in the treatment of certain B-cell blood cancers such as leukemia, lymphoma and multiple myeloma, this therapy still faces a major challenge: many patients will eventually relapse even after a short period of complete remission.
Recently, researchers from the Technical University of Munich in Germany published a research paper titled "Innate immune training restores pro-reparative myeloid functions to promote remyelination in the aged central nervous system" in the Cell journal Immunity. The study showed that age-related epigenomic changes impair the regenerative function of microglia, but innate immune training can reverse these changes and enhance their pro-regenerative ability in demyelinating injuries. These findings provide new insights into the aging-related decline of myeloid function and how to prevent this decline through innate immune reprogramming.
Breast cancer remains one of the most challenging cancers, mainly due to its heterogeneity and metastatic propensity. The Notch signaling pathway is required for multiple cellular processes and is involved in the development and progression of breast cancer. Noncoding RNAs (ncRNAs) have now become key regulators of gene expression, which can affect the biology of cancer by interacting with the Notch signaling pathway. Noncoding RNAs include microRNAs, long noncoding RNAs (lncRNAs), and circular RNAs (circRNAs). Recently, in a review report published in the international journal Gene Expression, entitled "Non-coding RNAs Affect Breast Cancer Development Through the Notch Signaling Pathway: An Overview", researchers from Tabriz University outlined the importance of ncRNAs in human breast cancer and their potential as therapeutic targets, paving the way for the development of innovative therapies to significantly impact patient care and prognosis.
Respiratory syncytial virus (RSV) causes a significant disease burden in the global population, with an estimated 33.1 million cases each year, and is the leading cause of bronchitis and viral pneumonia in infants. It particularly affects children and the elderly, and treatment options are limited and relatively ineffective.
Adeno-associated virus (AAV)-based viral vectors used in human gene therapy can induce innate immune pathways, leading to the initiation of the body's adaptive immune response. Recently, in a review article entitled "Innate Immune Sensing of Adeno-Associated Virus Vectors" published in the international journal Human Gene Therapy, scientists from Indiana University and other institutions described the range of possible redundant innate immune pathways that AAV vectors can activate, which will lead to excessive adaptive immune responses.
Hepatocellular carcinoma (HCC) is the most common type of primary liver cancer and is a highly lethal malignancy with a 5-year survival rate of less than 20%. Although targeted drugs such as sorafenib and other kinase inhibitors have been used to treat HCC, these therapies are generally incurable. Immunotherapy (e.g., atezolizumab) combined with vascular endothelial growth factor (VEGF) inhibition (bevacizumab) is currently the first-line treatment for HCC, but the efficacy remains low.
Gene replacement using adeno-associated virus (AAV) vectors is a promising approach to treat many diseases. However, the packaging capacity of AAV (about 4.7 kilobases) poses a challenge to this treatment modality, limiting its application in diseases associated with larger protein coding sequences (such as the 14 kilobases of mRNA in Duchenne muscular dystrophy).
Type I interferon (IFN-I) and IFN-γ can promote anti-tumor immunity by promoting the body's T cell response. Paradoxically, IFN-γ can promote T cell exhaustion by activating immune checkpoints, and the downstream regulatory mechanisms of these different responses are still unclear to researchers. Recently, in a research report entitled "Opposing tumor-cell-intrinsic and -extrinsic roles of the IRF1 transcription factor in antitumor immunity" published in the international journal Cell Reports, scientists from the David Geffen School of Medicine at the University of California and other institutions revealed the role and details of a special protein called interferon regulatory factor (IRF1) in cancer progression and response to therapy, which is expected to provide new insights to help improve the efficacy of cancer immunotherapy.
Recently, in a research report titled "Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence" published in the international journal Molecular Therapy-Methods & Clinical Development, scientists from the University of Wisconsin-Madison and other institutions have developed a new way to fight human cancer by studying T cells. Researchers said that this new therapy for treating blood cancer by using the power of the immune system to target and destroy cancer cells may be effective in treating human solid tumors.
