Results 41 to 50 of about 8,605,496 (403)

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

New England Journal of Medicine, 2019
Genome sequencing is often pivotal in the diagnosis of rare diseases, but many of these conditions lack specific treatments. We describe how molecular diagnosis of a rare, fatal neurodegenerative condition led to the rational design, testing, and ...
Jinkuk Kim, Chu Hu, Christelle Moufawad Achkar, L. Black, J. Douville, A. Larson, Mary K. Pendergast, S. F. Goldkind, E. Lee, E. Lee, Ashley Kuniholm, Aubrie Soucy, J. Vaze, Nandkishore R. Belur, Kristina Fredriksen, Iva Stojkovska, A. Tsytsykova, M. Armant, Renata L DiDonato, Jaejoon Choi, L. Cornelissen, L. M. Pereira, E. Augustine, C. Genetti, K. Dies, Brenda Barton, Brenda Barton, L. Williams, L. Williams, Benjamin D. Goodlett, Bobbie L Riley, A. Pasternak, E. Berry, Kelly A Pflock, Stephen Chu, C. Reed, K. Tyndall, P. Agrawal, A. Beggs, P. Grant, D. Urion, R. Snyder, S. Waisbren, A. Poduri, P. J. Park, Al Patterson, A. Biffi, Joseph R. Mazzulli, O. Bodamer, O. Bodamer, C. Berde, Timothy W. Yu, Timothy W. Yu   +52 more
semanticscholar   +1 more source

Patient and Disease-Specific Induced Pluripotent Stem Cells for Discovery of Personalized Cardiovascular Drugs and Therapeutics. [PDF]

, 2020
Human induced pluripotent stem cells (iPSCs) have emerged as an effective platform for regenerative therapy, disease modeling, and drug discovery.
Chandy, Mark, Paik, David T, Wu, Joseph C   +2 more
core  

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

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

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

Cellular and genetic therapies for haemophilia [PDF]

Haemophilia, 2006
Summary.  Haemophilia continues to be a prime target for a variety of gene and cell‐based therapies. Pre‐clinical successes in both mouse and dog models of the disease have been documented with a variety of approaches over the past decade, and there have now been six small clinical trials of gene transfer in haemophilia.
Lillicrap, David, VandenDriessche, T., High, K.   +2 more
openaire   +2 more sources

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

Cystic Fibrosis: New Trends in Therapy Methods

Архивъ внутренней медицины
This review provides information on recent advancements in the treatment of cystic fi brosis and presents interim results from ongoing clinical trials. Various scientifi c databases, including Scopus, Web of Science, and EMBASE, were utilized during the ...
P. A. Suchkova, S. A. Panova, O. Ya. Lisenko, K. P. Raevskij   +3 more
doaj   +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.
John J, Rossi, Carl H, June, Donald B, Kohn   +2 more
openaire   +2 more sources

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