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AAV Gene transfer - Crossref

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Last Updated: 16 May 2022

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Systemic Human Htert Aav Gene Transfer Therapy And The Effect On Telomere Length And Biological Age, A Case Report

A single adult female human was treated with AAV hTERT gene transfer therapy on two separate occasions, five years ago. A total intravenous dose of 3e15 AAV hTERT was administered on 9. 16. 2015. The second dose, which was administered on 9. 24. 2020, contained a total intravenous dose of 3e15 AAV hTERT. On 9. 15. 2015, the first telomere length measurements conducted at the start of the AAV hTERT gene transfer therapy reached a baseline average telomere length of 6. 71 kb, which corresponded to a telomere percentage adjusted to age and population at the 30th percentile. The most recent telomere length measurements, obtained on 7. 13. 2021, showed an average telomere length of 8. 94 kb, which corresponds to an age and population at the 89th percentile. Historically on 9. 15. 2015, and before the introduction of AAV hTERT gene transfer therapy, the associated age was estimated to be 62 years.

Source link: https://doi.org/10.37191/mapsci-2582-385x-4(2)-106


Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients

One of the main objectives of in vivo gene transfer is to develop therapeutic transgenes in terminally differentiated cells for long-term expression. A large number of AAV infections in the human race is now visible as a result of a lack, after being exposed to wild-type AAVs, anti-AAV antibodies develop and may neutralize the virus-derived vectors. Injection of AAV in humans is generally well tolerated, although the immune system may be activated after the identification of AAV vector capsid and genome. Since the first injection, the development of high-titer neutralizing antibodies to AAV has prevented vector re-administration. Clinical translation and demonstration of the safety and effectiveness of these techniques, as well as providing a curative therapy to patients are largely due to extensive preclinical research and the large clinical experience in the surveillance of AAV vectors immunogenicity.

Source link: https://doi.org/10.3389/fimmu.2022.857276


Therapeutic Applications of Adeno-Associated Virus (AAV) Gene Transfer of HLA-G in the Eye

The aim of this paper is to investigate human leukocyte antigen G in the eye, its role in immune tolerance, and the potential therapeutic use of AAV gene transfer and expression of HLA-G in various ocular tissues.

Source link: https://doi.org/10.3390/ijms23073465


Abstract 170: Exosomal AAV-mediated SERCA2a Gene Transfer Improves Cardiac Function in a Mouse Model of Heart Failure

However, pre-existing antibodies to several cardiotropic AAV serotypes naturally present in humans pose a significant obstacle for gene therapy translation to clinical applications. We use exosomal AAVs as a reliable transduction device that also shields pre-existing immunity to the viral capsid. In vitro and in a passive immunity nude mouse model in vivo, efficiency of heart targeting was then assessed for eAAV9 or eAAV6 and standard AAV9 or AAV6 in human cardiomyocytes. In addition, we found in vivo that eAAV9-mediated gene delivery is more specific to CMs than to other cell types present in the heart, indicating that eAAVs maintain the AAV9 serotype's cardiotropic characteristics. Our preliminary findings showed that eAAV infectious entry could involve trafficking through endocytic compartments as a result of colocalization of eAAVs and markers specific for endosomes in vitro.

Source link: https://doi.org/10.1161/res.125.suppl_1.170


Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer

In studies with AAV5-FVIII-SQ, an investigational gene therapy for hemophilia A, residual vector DNA was detectable in blood, secrecy, and excreta, but it was also unknown how long AAV5 vector capsids were present, but it was unclear how long structurally intact AAV5 vector capsids were present. In plasma and semen, respectively, the detection range for AAV5 vector capsids was 1. 17E+04 and 2. 33E+04 vg/mL. AAV5 capsid quantification was reduced by 141 ng/mL, suggesting that iqPCR mainly detects free capsids rather than those that are complicated with antibodies.

Source link: https://doi.org/10.1038/s41434-021-00287-1


Intravenous gene transfer throughout the brain of infant Old World primates using AAV

Although the scientific community has produced numerous neurotropic AAV variants for systemic gene transfer to the rodent brain, there are no AAVs that accesses Primates' CNS. The rhesus macaque and the green monkey are two common infants of two Old World primate species, AAV. CAP-Mac, an engineered AAV variant that allows systemic, brain-wide gene delivery in infants of two endangered species, AAV. CAP-Mac. We'll talk about AAV. CAP-Mac, an engineered AAV variant that allows systemic, brain-wide gene delivery in infants of two Old World primate species (the rhes We developed CAP-Mac with a multi-species selection process, first screening our library in the adult common marmoset and narrowing our pool of test-variants for another round of selection in infant macaques. CAP-Mac reliably transduces human neurons in vitro and Old World primate neurons in vivo, where it affects all lobes of cortex, cerebellum, and multiple subcortical areas of disease relevance in individual characterization. Both medium spiny neurons and cortical pyramidal cells can be restored morphologically by using CAP-Mac for Brainbow-like multicolor labeling of macaque neurons throughout the brain, enabling the morphological reconstruction of both medium spiny neurons and cortical pyramidal cells.

Source link: https://doi.org/10.1101/2022.01.08.475342

* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions

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* Please keep in mind that all text is summarized by machine, we do not bear any responsibility, and you should always check original source before taking any actions