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Adeno-associated virus as a delivery vector for gene therapy of human diseases
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to...
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An optimized protocol for the generation and monitoring of conditional orthotopic lung cancer in the KP mouse model using an adeno-associated virus vector compatible with biosafety level 1
BackgroundThe inducible Kras/p53 lung adenocarcinoma mouse model, which faithfully recapitulates human disease, is routinely initiated by the...
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Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
Purpose of ReviewRecombinant adeno-associated virus (rAAV) is the commonest viral vector used in gene therapies. With the increase in the number of...
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Current Advances in Adeno-Associated Virus-Mediated Gene Therapy to Prevent Acquired Hearing Loss
Adeno-associated viruses (AAVs) are viral vectors that offer an excellent platform for gene therapy due to their safety profile, persistent gene...
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A 360-degree perspective on adeno-associated virus (AAV)-based gene therapy for haemophilia: Insights from the physician, the nurse and the patient
BackgroundAdeno-associated virus (AAV)-based gene therapy for haemophilia has advanced substantially in the last 13 years; recently, three products...
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Adeno-associated virus (AAV) 9-mediated gene delivery of Nurr1 and Foxa2 ameliorates symptoms and pathologies of Alzheimer disease model mice by suppressing neuro-inflammation and glial pathology
There is a compelling need to develop disease-modifying therapies for Alzheimer’s disease (AD), the most common neuro-degenerative disorder. Together...
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Construction of Tissue-engineered Cartilage In Vivo from Microtia Chondrocytes After Transfection with Human VEGF165 Genes Mediated by a Recombinant Adeno-Associated Viral Vector
ObjectiveTo evaluate the transfection efficiency of cultured chondrocytes from individuals with microtia (microtia chondrocytes) with the recombinant...
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Protocol: Adeno-Associated Virus-Mediated Gene Transfer in Ex Vivo Cultured Embryonic Mammary Gland
Branching morphogenesis of the murine mammary gland starts during late embryogenesis. It is regulated by the signals emanating both from the...
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A Kaposi’s sarcoma-associated herpes virus-encoded microRNA contributes to dilated cardiomyopathy
Dilated cardiomyopathy (DCM) is the leading cause of heart transplantation. By microRNA (miRNA) array, a Kaposi’s sarcoma-associated herpes virus...
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Host cell factors involved in classical swine fever virus entry
Classical swine fever virus (CSFV) is an ancient pathogen that continues to pose a threat to animal agriculture worldwide. The virus belongs to the...
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Optogenetic activation of the lateral habenulaD1R–ventral tegmental area circuit induces depression-like behavior in mice
A number of different receptors are distributed in glutamatergic neurons of the lateral habenula (LHb). These glutamatergic neurons are involved in...
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Impact of COVID‑19 pandemic restrictions and subsequent relaxation on the prevalence of respiratory virus hospitalizations in children
BackgroundThe COVID-19 pandemic and the consequently adopted worldwide control measures have resulted in global changes in the epidemiology and...
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Respiratory illness virus infections with special emphasis on COVID-19
Viruses that emerge pose challenges for treatment options as their uniqueness would not know completely. Hence, many viruses are causing high...
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Etranacogene Dezaparvovec: First Approval
Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; Hemgenix ® ) is an adeno-associated virus vector-based gene therapy being developed by...
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Gene therapy for liver diseases — progress and challenges
Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential for treating and curing genetic disorders. For otherwise...
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Valoctocogene Roxaparvovec: First Approval
Valoctocogene roxaparvovec (ROCTAVIAN ™ ) is a gene therapy being developed by BioMarin Pharmaceutical Inc. for the treatment of haemophilia A. In...
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Adeno-associated virus as a gene therapy vector: strategies to neutralize the neutralizing antibodies
Adeno-associated virus (AAV)-derived vectors are currently the most common type of viral vectors used in gene therapy clinical trials. The presence...
