Results 41 to 50 of about 8,660,824 (403)
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 +1 more
doaj +1 more source
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 +9 more
doaj +1 more source
Advances in the design and development of oncolytic measles viruses. [PDF]
, 2015 A successful oncolytic virus is one that selectively propagates and destroys cancerous tissue without causing excessive damage to the normal surrounding tissue. Oncolytic measles virus (MV) is one such virus that exhibits this characteristic and thus has
Hutzen, Brian +2 more
core +1 more source
Cystic Fibrosis: New Trends in Therapy Methods
Архивъ внутренней медициныThis 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 +3 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 +2 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 +11 more
semanticscholar +1 more source
CRISPR/Cas9 mediated knockout of rb1 and rbl1 leads to rapid and penetrant retinoblastoma development in Xenopus tropicalis [PDF]
, 2016 Retinoblastoma is a pediatric eye tumor in which bi-allelic inactivation of the Retinoblastoma 1 (RB1) gene is the initiating genetic lesion. Although recently curative rates of retinoblastoma have increased, there are at this time no molecular targeted ...
Boel, Annekatrien +12 more
core +2 more sources
Circulation, 2019 Supplemental Digital Content is available in the text. Background: The ability of a genetic risk score to predict risk in established cardiovascular disease and identify individuals who derive greater benefit from PCSK9 (proprotein convertase subtilisin ...
N. Marston +14 more
semanticscholar +1 more source
Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
core +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 +3 more
doaj +1 more source