Results 61 to 70 of about 8,933,868 (252)

Modulation of miR‐23b Wnt/β‐catenin Axis Strengthens Endothelial Barrier Properties

Advanced Science, EarlyView.
Early blood‐brain barrier (BBB) disruption contributes to stroke and CNS disease pathology. miR‐23b was identified as a regulator of BBB integrity in brain endothelial cells. Inhibition of miR‐23b enhanced barrier‐associated properties, promoted repair‐related signaling, and reduced BBB leakage in experimental stroke models, supporting further ...
Victor Anthony Martinez, Rose E. Presby, Sara Nakanishi, Pallavi Gaur, Ugur Akcan, Johannes Daugaard Krog, Iben Daugaard Krog, Lipi Das, David S. Johnson, Danny Jamoul, Douglas Jury, Brian Lawson, Jacob Körbelin, Lewis M. Brown, Vilas Menon, Dritan Agalliu, Irene Munk Pedersen   +16 more
wiley   +1 more source

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Hanawa, H, Ng, CYC, Mandrell, TD, Vanin, EF, Nathwani, AC, Hu, Y, Davidoff, AM, Hoffer, FA, Zhou, J, Slaughter, C, Nikanorov, A, Lozier, JN, Nienhuis, AW   +12 more
core  

Image_1_Co-transduction of dual-adeno-associated virus vectors in the neonatal and adult mouse utricles.TIF

, 2022
Adeno-associated virus (AAV)-mediated gene transfer is an efficient method of gene over-expression in the vestibular end organs. However, AAV has limited usefulness for delivering a large gene, or multiple genes, due to its small packaging capacity ...
Jing-Ying Guo (743732), Zhong-Rui Chen (11848124), Jun-Yi Xu (13980396), Shu-Sheng Gong (743733), Zi-Jing Yang (13980399), Ke Liu (121889), Wei Su (89824), Shan Liu (193759), Lu He (811687), Guo-Peng Wang (743731)   +9 more
core   +1 more source

Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

Viruses
Today, adeno-associated virus (AAV)-based vectors are arguably the most promising in vivo gene delivery vehicles for durable therapeutic gene expression. Advances in molecular engineering, high-throughput screening platforms, and computational techniques
Estrella Lopez-Gordo, Kyle Chamberlain, Jalish Mahmud Riyad, Erik Kohlbrenner, Thomas Weber   +4 more
doaj   +1 more source

AAV vectors accumulate in the pineal gland after injections into the brain or spinal cord

Molecular Therapy: Methods & Clinical Development, 2021
AAV vectors are being used extensively for gene-modifying therapies for neurological disorders. Here, we report the surprising discovery that injections of different AAVs into the brain, spinal cord, or cerebrospinal fluid (CSF) lead to robust ...
Oswald Steward, Aminata P. Coulibaly, Mariajose Metcalfe, Jamie M. Dam, Kelly M. Yee   +4 more
doaj   +1 more source

BAP31 Drives Cartilage Calcification through Disruption of Autophagosome–Lysosome Fusion in Osteoarthritis

Advanced Science, EarlyView.
Mechanical stress activates BAP31 in chondrocytes. BAP31 competes with ATG14 for binding to STX17, disrupting the STX17–ATG14 complex required for autophagosome–lysosome fusion. The resultant autophagic flux blockade drives the generation of autophagy‐derived exosomes, which mediate pathological cartilage calcification in OA. Chondrocyte‐targeted BAP31
Zhi‐hua Xu, Ruo‐xin Wang, Feng He, Qian Liu, Fu‐yin Li, Min Hui, Yu‐qian Shi, Yu Jiang, Qing‐hua Li, Jun‐wen Jing, Shi‐bin Yu, Li‐tian Ma, Qian‐nan Niu, Mian Zhang   +13 more
wiley   +1 more source

The role of the coactivators PGC-1α and PGC-1β in retina and skeletal muscle [PDF]

, 2011
Peroxisome proliferator-activated receptor γ coactivator- 1 (PGC-1) designates a family of coactivators consisting of PGC-1α, PGC-1β and PRC. By associating with transcription factors like nuclear receptors, they induce transcription of target genes that
Egger, A, Egger, Anna Franziska
core   +1 more source

AAV-based vectors for human diseases modeling in laboratory animals

Frontiers in Medicine
The development of therapeutic drugs and vaccines requires the availability of appropriate model animals that replicate the pathogenesis of human diseases. Both native and transgenic animals can be utilized as models.
Timur I. Aliev, Dmitry V. Yudkin
doaj   +1 more source

Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV

Advanced Science, EarlyView.
Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes, Gabriela Silva, Mafalda Aleixo, Marta Gomes, Daniel Simão, Stephan J. Holtkamp, Diana D. Lobo, Pradeep Harish, Rosalind Jenkins, Lekh N. Dahal, Rui J. Nobre, Luís de Pereira de Almeida, Mark Trautwein, Paula M. Alves, Catarina Brito   +14 more
wiley   +1 more source

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Hanawa, H, Vanin, EF, Nathwani, AC, Zhou, JF, Davidoff, A, Nienhuis, AW   +5 more
core