Date:
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  • 24-01-11
  • Virus
  • Sciences

Advantages And Challenges of Clinical Application of AAV Gene Therapy

Gene therapy is one of the hottest therapeutic fields at present, and multiple gene therapies have been approved by regulatory agencies in recent years. A number of pipelines that have entered late-stage clinical studies are also approaching approval for marketing. At present, most gene therapy applications still use viral vectors. These viral vectors have been modified so that they are no longer pathogenic. Widely used viral vectors include adenoviral vectors (ADV), lentiviral vectors (LV), herpes simplex virus vectors (HSV), poxvirus vectors (PV) and adeno-associated virus vectors (AAV).

  • 24-01-10
  • Virus
  • Sciences

AAV Vector Optimization Strategy: Enhancing Infection Efficiency And Targeting

AAV has broad application prospects as a vector. In addition to being used for the treatment of genetic diseases, it can be used for the treatment of tumors and can also be used as a vector to construct vaccines. AAV also has certain applications in influenza prevention. AAV-mediated antibody expression can protect elderly and immunodeficient mice from influenza virus damage.

  • 23-11-30
  • Virus
  • New Research

Integration of AAV Viruses Into the Human Genome Does Not Cause Cancer But Results In Durable Expression

Professor James Wilson's team from the University of Pennsylvania published a research paper titled "Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration" in the journal Nature Biotechnology. On the same day, Professor James Wilson's team also published a research paper titled "Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans" in the journal Human Gene Therapy.

  • 23-09-01
  • Gene / Virus
  • New Research

AAV Gene Therapy Significantly Reduces Alcohol Consumption in Heavy Drinking Nonhuman Primates

Alcohol use disorders (AUD) impose significant personal, social, and economic costs worldwide. Return to drinking is common among patients with AUD seeking treatment, resulting from cycles of repeated abstinence-relapse episodes, even with currently available pharmacotherapy.

  • 23-06-27
  • Virus
  • New Research

Stable Expression of Large Transgenes Achieved by Knock-in of Integrase-deficient Lentivirus

In order to maximize the therapeutic potential and improve the efficacy and safety of engineered cells, it is often necessary to express large fragments of genes or complex gene circuits in cells. For example, when constructing CAR-T cells, the ability of T cells to kill cancer cells can be improved by introducing chimeric antigen receptor (CAR) genes into T cells. CRISPR activation (CRISPRa), CRISPR interference (CRISPRi), and logic gates can further enhance the efficacy of CAR-T cell therapy, but its long-term expression in primary cells remains a major challenge.

  • 23-06-27
  • Cytology / Virus
  • Sciences

Scientists Identify Special Protein That Helps HIV Stay Silent

Antiretroviral therapy (ART) has transformed HIV-1 infection into a manageable chronic disease and increased the life expectancy of people living with HIV-1 (PLWH). During antiretroviral therapy, HIV can quietly hide in a reservoir of CD4+ T lymphocytes, a special type of white blood cell that plays an important role in activating the body's immune system to fight infection. The existence of these viral sanctuaries may explain why antiretroviral therapy must be continued throughout a patient's life to prevent HIV from replicating. According to WHO data, by the end of 2021, more than 3,800 people worldwide will be infected with HIV.

  • 23-06-01
  • Cytology / Virus
  • New Research

New AAV System Once Again Confirms That NeuroD1 Cannot Mediate Neuronal Transdifferentiation In Vivo

Research On Glial Cells

  • 20-04-07
  • Virus
  • Report

Efficient Creation of Oncolytic Viruses Using the CRISPR-Cas9 System

Oncolytic viruses (OVs) are natural or recombinant viruses that prefer to infect cancer cells. This feature has made them one of the main current research topics on cancer therapeutics and the significant element in the future of cancer treatment. Over the past two decades, there has been growing evidence that OVs are effective in treating cancer in both preclinical models and clinical trials. The most tested OVs in preclinical and clinical trials include the Herpes simplex virus (HSV), adenovirus (AdV) and Vaccinia virus (VV). In one study, an oncolytic HSV (T-VEC) coding for granulocyte/macrophage-colony stimulating factor (GM-CSF) was administered by direct intratumoral injection to patients with metastatic malignant melanoma and this resulted in complete regressions of injected and uninjected lesions in eight of 50 patients. In addition to the single therapy, T-VEC has also been used in combination with radiotherapy and cisplatin in clinical trials to treat stage III/IV head and neck cancer. An oncolytic vaccinia virus (JX-594) armed with GM-CSF, showed hopeful results in preclinical and clinical trials treating liver cancers.

  • 20-03-17
  • Virus
  • Report

How Monkeys, Mice and Ferrets Help Scientists Develop Potential Drugs and Vaccines for New Coronavirus Therapy

The construction of an animal model of new coronavirus can not only help us understand the pathological characteristics of the disease, but also help the development of drugs and vaccines. How do monkeys, mice and ferrets help scientists study new coronavirus? Recently, The magazine of Nature interviewed several scientists to understand their choices and opinions on different animal models.

  • 20-03-05
  • Virus
  • New Research

Major Progress! Reveal the Mechanism of SARS-CoV-2 Entry Into Host Cells

Several members of the Coronaviridae family continue to spread through the population and usually cause mild respiratory diseases. In contrast, severe acute respiratory syndrome coronavirus (SARS-CoV) and Middle East respiratory syndrome coronavirus (MERS-CoV) are transmitted from animals to humans and cause severe respiratory diseases SARS and MERS in patients, respectively. SARS appeared in Guangdong, China in 2002, and subsequently spread globally, causing 8096 cases and 774 deaths. Intermediate hosts such as civets promote transmission in humans. Currently, there are no antiviral drugs or approved vaccines to treat SARS, and the SARS epidemic of 2002-2003 was finally stopped through routine control measures, including travel restrictions and patient isolation.