Results 11 to 20 of about 1,217,743 (262)

Antibody-based CCR5 blockade protects Macaques from mucosal SHIV transmission

Nature Communications, 2021
CCR5 is a co-receptor for many transmitted HIV strains. Here, the authors show that biweekly injection of the CCR5-specific antibody Leronlimab protects rhesus macaques against infection following repeated intrarectal challenges of a CCR5-tropic SHIV.
Xiao L. Chang, Gabriela M. Webb, Helen L. Wu, Justin M. Greene, Shaheed Abdulhaqq, Katherine B. Bateman, Jason S. Reed, Cleiton Pessoa, Whitney C. Weber, Nicholas Maier, Glen M. Chew, Roxanne M. Gilbride, Lina Gao, Rebecca Agnor, Travis Giobbi, Jeffrey Torgerson, Don Siess, Nicole Burnett, Miranda Fischer, Oriene Shiel, Cassandra Moats, Bruce Patterson, Kush Dhody, Scott Kelly, Nader Pourhassan, Diogo M. Magnani, Jeremy Smedley, Benjamin N. Bimber, Nancy L. Haigwood, Scott G. Hansen, Timothy R. Brown, Lishomwa C. Ndhlovu, Jonah B. Sacha   +32 more
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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Nature Communications, 2020
Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and ...
Hung-Lun Hsu, Alexander Brown, Anna B. Loveland, Anoushka Lotun, Meiyu Xu, Li Luo, Guangchao Xu, Jia Li, Lingzhi Ren, Qin Su, Dominic J. Gessler, Yuquan Wei, Phillip W. L. Tai, Andrei A. Korostelev, Guangping Gao   +14 more
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Gene Therapy

Hematology/Oncology Clinics of North America, 2017
Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Barb, Thorne, Ryan, Takeya, Francesca, Vitelli, Xin, Swanson   +3 more
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MicroRNA-7, synergizes with RORα, negatively controls the pathology of brain tissue inflammation

Journal of Neuroinflammation, 2020
Background Accumulating evidence has documented that microRNA-7 (miR-7) plays an important role in the pathology of various diseases. However, the potential role of miR-7 in brain tissue inflammation (BTI) remains unclear.
Dongxu Yue, Juanjuan Zhao, Huizi Chen, Mengmeng Guo, Chao Chen, Ya Zhou, Lin Xu   +6 more
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Gene therapy. [PDF]

Gut, 1982
Gene therapy is not yet possible, but may become feasible soon, particularly for well understood gene defects. Although treatment of a patient raises no ethical problems once it can be done well, changing the genes of an early embryo is more difficult, controversial and unlikely to be required clinically.
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Human Cytomegalovirus Induces Cellular and Humoral Virus-specific Immune Responses in Humanized BLT Mice

Scientific Reports, 2017
The strict species specificity of Human Cytomegalovirus (HCMV) has impeded our understanding of antiviral adaptive immune responses in the context of a human immune system.
Lindsey B. Crawford, Rebecca Tempel, Daniel N. Streblow, Craig Kreklywich, Patricia Smith, Louis J. Picker, Jay A. Nelson, Patrizia Caposio   +7 more
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Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis

Skeletal Muscle, 2017
Background Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant regeneration in muscle.
Mehmet E. Yalvac, Jakkrit Amornvit, Cilwyn Braganza, Lei Chen, Syed-Rehan A. Hussain, Kimberly M. Shontz, Chrystal L. Montgomery, Kevin M. Flanigan, Sarah Lewis, Zarife Sahenk   +9 more
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Data collection infrastructure for patient outcomes in the UK – opportunities and challenges for cell and gene therapies launching

Journal of Market Access & Health Policy, 2019
Background: Cell and gene therapies are associated with uncertainty around their value claims at launch due to limitations of supporting clinical data; furthermore, their high costs present affordability issues for payers.
Jesper Jørgensen, Laura Mungapen, Panos Kefalas   +2 more
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Functional roles of the membrane-associated AAV protein MAAP

Scientific Reports, 2021
With a limited coding capacity of 4.7 kb, adeno-associated virus (AAV) genome has evolved over-lapping genes to maximise the usage of its genome. An example is the recently found ORF in the cap gene, encoding membrane-associated accessory protein (MAAP),
Lionel Galibert, Amira Hyvönen, Reetta A. E. Eriksson, Salla Mattola, Vesa Aho, Sami Salminen, Justin D. Albers, Sanna K. Peltola, Saija Weman, Tiina Nieminen, Seppo Ylä-Herttuala, Hanna P. Lesch, Maija Vihinen-Ranta, Kari J. Airenne   +13 more
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Cochlear Gene Therapy [PDF]

Cold Spring Harbor Perspectives in Medicine, 2012
Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss.
Lawrence R, Lustig, Omar, Akil
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