Results 21 to 30 of about 835,919 (314)

AAV8 locoregional delivery induces long-term expression of an immunogenic transgene in macaques despite persisting local inflammation

Molecular Therapy: Methods & Clinical Development, 2021
Adeno-associated virus (AAV) vectors are considered efficient vectors for gene transfer, as illustrated by recent successful clinical trials targeting retinal or neurodegenerative disorders.
Gwladys Gernoux, Mickaël Guilbaud, Marie Devaux, Malo Journou, Virginie Pichard, Nicolas Jaulin, Adrien Léger, Johanne Le Duff, Jack-Yves Deschamps, Caroline Le Guiner, Philippe Moullier, Yan Cherel, Oumeya Adjali   +12 more
doaj  

A Case of a Rapidly Enlarging Neck Mass with Airway Compromise [PDF]

Journal of Clinical and Diagnostic Research, 2017
Anaplastic Thyroid Carcinoma (ATC) is one of the most lethal tumours in humans, extremely rare in occurrence and very aggressive in nature. We hereby present a rare case of ATC with airway compromise.
Libardo Rueda Prada, Venkata Sandeep Koripalli, Cesar Luis Merino, Ilmana Fulger   +3 more
doaj   +1 more source

Genetics and the Individualized Therapy of Vestibular Disorders [PDF]

Frontiers in Neurology, 2021
Background: Vestibular disorders (VDs) are a clinically divergent group of conditions that stem from pathology at the level of the inner ear, vestibulocochlear nerve, or central vestibular pathway. No etiology can be identified in the majority of patients with VDs.
Christine Mei, Hongsong Dong, Hongsong Dong, Eric Nisenbaum, Torin Thielhelm, Aida Nourbakhsh, Denise Yan, Molly Smeal, Yesha Lundberg, Michael E. Hoffer, Simon Angeli, Fred Telischi, Guohui Nie, Susan H. Blanton, Xuezhong Liu   +14 more
openaire   +3 more sources

Genetic therapies against HIV [PDF]

Nature Biotechnology, 2007
Highly active antiretroviral therapy prolongs the life of HIV-infected individuals, but it requires lifelong treatment and results in cumulative toxicities and viral-escape mutants. Gene therapy offers the promise of preventing progressive HIV infection by sustained interference with viral replication in the absence of chronic chemotherapy.
Donald B. Kohn, John J. Rossi, Carl H. June   +2 more
openaire   +3 more sources

Genetics and therapy for pediatric eye diseases [PDF]

EBioMedicine, 2021
Ocular morphogenesis in vertebrates is a highly organized process, orchestrated largely by intrinsic genetic programs that exhibit stringent spatiotemporal control. Alternations in these genetic instructions can lead to hereditary or nonhereditary congenital disorders, a major cause of childhood visual impairment, and contribute to common late-onset ...
Holly.Y. Chen, Ordan J. Lehmann, Anand Swaroop   +2 more
openaire   +4 more sources

The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs

Molecular Therapy: Methods & Clinical Development, 2022
Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect.
Yuji Kashiwakura, Nemekhbayar Baatartsogt, Shoji Yamazaki, Azusa Nagao, Kagehiro Amano, Nobuaki Suzuki, Tadashi Matsushita, Akihiro Sawada, Satoshi Higasa, Naoya Yamasaki, Teruhisa Fujii, Taemi Ogura, Hideyuki Takedani, Masashi Taki, Takeshi Matsumoto, Jun Yamanouchi, Michio Sakai, Masako Nishikawa, Yutaka Yatomi, Koji Yada, Keiji Nogami, Ryota Watano, Takafumi Hiramoto, Morisada Hayakawa, Nobuhiko Kamoshita, Akihiro Kume, Hiroaki Mizukami, Shizukiyo Ishikawa, Yoichi Sakata, Tsukasa Ohmori   +29 more
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Assessment of the frequency and association with morbidity of DNA markers in multinational administrative divisions based on indigenous population data (based on cardiovascular diseases)

Кардиоваскулярная терапия и профилактика
Information on morbidity is presented in statistical reports for the entire population of multinational subjects of Russia, but population Biobanks contain information on individual peoples.Aim.
E. V. Balanovskaya, A. Yu. Potanina, S. M. Koshel, D. S. Adamov, I. V. Evsyukov, A. L. Borisova, M. S. Pokrovskaya, A. N. Meshkov   +7 more
doaj   +1 more source

Graft Materials and Biologics for Spinal Interbody Fusion

Biomedicines, 2019
Spinal fusion is the most widely performed procedure in spine surgery. It is the preferred treatment for a wide variety of pathologies including degenerative disc disease, spondylolisthesis, segmental instability, and deformity.
Marissa D’Souza, Nicholas A. Macdonald, Julian L. Gendreau, Pate J. Duddleston, Austin Y. Feng, Allen L. Ho   +5 more
doaj   +1 more source

Prenatal diagnosis and genetic etiology analysis of talipes equinovarus by chromosomal microarray analysis

BMC Medical Genomics, 2023
Background With the advancement of molecular technology, fetal talipes equinovarus (TE) is believed to be not only associated with chromosome aneuploidy, but also related to chromosomal microdeletion and microduplication.
Xiaorui Xie, Baojia Huang, Linjuan Su, Meiying Cai, Yuqin Chen, Xiaoqing Wu, Liangpu Xu   +6 more
doaj   +1 more source

Genetic therapy for the nervous system [PDF]

Human Molecular Genetics, 2011
Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising.
William J. Bowers, Xandra O. Breakefield, Miguel Sena-Esteves   +2 more
openaire   +2 more sources