Results 11 to 20 of about 6,300,245 (266)

Update on Viral Gene Therapy Clinical Trials for Retinal Diseases [PDF]

, 2022
In 2001, the first large animal was successfully treated with a gene therapy that restored its vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness called Leber's congenital amaurosis type 2.
Cheng, Shun-Yun, Punzo, Claudio
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Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles [PDF]

, 2022
Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease, in which soft connective tissue is converted into heterotopic bone through an endochondral ossification process.
Shim, Jae-Hyuck, Delai, Patricia L R, Madhuri, Vrisha, De Cunto, Carmen L, de Ruiter, Ruben D, Schoenmaker, Ton, Zasloff, Michael, Friedman, Clive S, Gao, Guangping, Cho, Tae-Joon, Morhart, Rolf, Grunwald, Zvi, di Rocco, Maja, Haga, Nobuhiko, de Vries, Teun J, Hsiao, Edward C, Pals, Gerard, Netelenbos, Coen, Diecidue, Robert J, Kaplan, Frederick, Smilde, Bernard J, Scott, Christiaan, Eekhoff, Elisabeth M W, Pignolo, Robert J, Micha, Dimitra   +24 more
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Durability of transgene expression after rAAV gene therapy [PDF]

, 2022
Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the lives of patients with certain genetic disorders by increasing or restoring function to affected tissues.
Schulz, Martin, Levy, Daniel I, McCarty, Douglas, Gao, Guangping, Muhuri, Manish   +4 more
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Editorial: "AAV Gene Therapy: Immunology and Immunotherapeutics" [PDF]

, 2021
Editorial on the Research Topic: AAV Gene Therapy: Immunology and ...
Gao, Guangping, Paulk, Nicole K, Martinez-Navio, José M   +2 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
doaj   +1 more source

Emerging Concepts and Challenges in Rheumatoid Arthritis Gene Therapy

, 2020
Rheumatoid arthritis (RA) is a systemic inflammatory joint disease affecting about 1% of the population worldwide. Current treatment approaches do not ensure a cure for every patient.
Ludmila V. Akhmadishina, Marina I. Beloukhova, Alexander N. Lukashev, Vadim V. Tarasov, Yulia A. Vakulenko, Andrey A. Zamyatnin Jr., Andrei A. Deviatkin   +6 more
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Cystic Fibrosis gene therapy in the UK and elsewhere [PDF]

, 2015
The cystic fibrosis transmembrane conductance regulator (CFTR) gene was identified in 1989. This opened the door for the development of cystic fibrosis (CF) gene therapy, which has been actively pursued for the last 20 years.
Griesenbach, U, Pytel, K, Alton, EWFW
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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
doaj   +1 more source

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
doaj   +1 more source

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
doaj   +1 more source