Results 31 to 40 of about 7,750,425 (359)

Impact of novel hemophilia therapies around the world

Research and Practice in Thrombosis and Haemostasis, 2022
Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard‐of‐care approach in hemophilia.
Margareth C. Ozelo, Gabriela G. Yamaguti‐Hayakawa   +1 more
doaj   +1 more source

Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders

Frontiers in Genetics, 2019
The field of gene therapy is striving more than ever to define a path to the clinic and the market. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide.
Alireza Shahryari, Marie saghaeian Jazi, S. Mohammadi, Hadi Razavi Nikoo, Zahra Nazari, Elaheh Sadat Hosseini, I. Burtscher, S. Mowla, H. Lickert   +8 more
semanticscholar   +1 more source

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

Evaluation of the Autologous Genetically Enriched Leucoconcentrate on the Lumbar Spinal Cord Morpho-Functional Recovery in a Mini Pig with Thoracic Spine Contusion Injury

Biomedicines, 2023
Background: Pathological changes associated with spinal cord injury (SCI) can be observed distant, rostral, or caudal to the epicenter of injury. These remote areas represent important therapeutic targets for post-traumatic spinal cord repair.
Ravil Garifulin, Maria Davleeva, Andrei Izmailov, Filip Fadeev, Vage Markosyan, Roman Shevchenko, Irina Minyazeva, Tagir Minekayev, Igor Lavrov, Rustem Islamov   +9 more
doaj   +1 more source

Genetic Variants Associated With Cancer Therapy–Induced Cardiomyopathy

Circulation, 2019
Supplemental Digital Content is available in the text.
P. García-Pavía, Yuri Kim, M. A. Restrepo-Córdoba, I. Lunde, H. Wakimoto, A. Smith, Christopher N Toepfer, K. Getz, J. Gorham, Parth N. Patel, Kaoru Ito, J. A. Willcox, Z. Arany, Jian Li, A. Owens, R. Govind, B. Nuñez, Erica Mazaika, A. Bayés‐Genís, R. Walsh, B. Finkelman, J. Lupón, N. Whiffin, I. Serrano, W. Midwinter, Alicja E Wilk, A. Bardají, N. Ingold, R. Buchan, U. Tayal, D. Pascual-Figal, A. de Marvao, Mian Ahmad, J. M. García-Pinilla, A. Pantazis, F. Domínguez, A. John Baksi, D. O’Regan, S. Rosen, S. Prasad, E. Lara-Pezzi, M. Provencio, A. Lyon, L. Alonso-Pulpón, S. Cook, S. DePalma, P. Barton, R. Aplenc, J. Seidman, B. Ky, J. Ware, C. Seidman   +51 more
semanticscholar   +1 more source

Management of genetic diseases: Present and future

Revista de la Facultad de Medicina Humana, 2021
Today, the number of genetic diseases is around 10000 conditions, affecting to 6%-8% of all populations. This review shows us how the discovery of genetic variants in our genome, this facilitated to know with precision about the mechanisms ...
Hugo Hernán Abarca Barriga, Milana Trubnykova, María del Carmen Castro Mujica   +2 more
doaj   +1 more source

Beta-Ketothiolase Deficiency: A Comprehensive Review of Genetic Variants and Pathophysiology [PDF]

Annals of Child Neurology
Beta-ketothiolase deficiency (BKD) is a rare autosomal recessive disorder caused by mutations in the ACAT1 gene, also known as mitochondrial acetoacetyl-coenzyme A thiolase (MAT) deficiency.
Sohit Kashyap, Anil Kumar, Anita Choudhary, Ajay Kumar, Arvinder Wander, Anjana Munshi   +5 more
doaj   +1 more source

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

Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders

Nature Communications, 2019
Fifty percent of inner ear disorders are caused by genetic mutations. To develop treatments for genetic inner ear disorders, we designed gene replacement therapies using synthetic adeno-associated viral vectors to deliver the coding sequence for ...
Carl Nist-Lund, B. Pan, A. Patterson, Y. Asai, Tianwen Chen, Wu Zhou, Hong Zhu, Sandra Romero, Jennifer Resnik, D. Polley, G. Géléoc, J. R. Holt   +11 more
semanticscholar   +1 more source

DNA Repair Pathways in Cancer Therapy and Resistance

Frontiers in Pharmacology, 2021
DNA repair pathways are triggered to maintain genetic stability and integrity when mammalian cells are exposed to endogenous or exogenous DNA-damaging agents.
Lan-ya Li, Yi-di Guan, Xi-sha Chen, Jin-Ming Yang, Yan Cheng   +4 more
semanticscholar   +1 more source