Results 61 to 70 of about 9,448,988 (355)
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
Gene Therapy – Potential, Pros, Cons and Ethics [PDF]
, 2002 Genetic technology poses risks along with its rewards, just as any technology has in the past. To stop its development and forfeit the benefits gene therapy could offer would be a far greater mistake than forging ahead could ever be.
Rao Nanjunda, Mr Ananth
core
Clinical application of high throughput molecular screening techniques for pharmacogenomics. [PDF]
, 2011 Genetic analysis is one of the fastest-growing areas of clinical diagnostics. Fortunately, as our knowledge of clinically relevant genetic variants rapidly expands, so does our ability to detect these variants in patient samples.
Schrijver, Iris, Wiita, Arun P
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Purpose Retinoblastoma (RB) is the most common pediatric ocular cancer, yet population‐based data on survival and risk factors remain limited. This study aimed to describe survival in a large national RB cohort and identify predictors of death and complications.
Samuel Sassine +14 more
wiley +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
Messenger RNA therapy for rare genetic metabolic diseases
Gut, 2019 Decades of intense research in molecular biology and biochemistry are fructifying in the emergence of therapeutic messenger RNAs (mRNA) as a new class of drugs.
P. Berraondo +3 more
semanticscholar +1 more source
AAV vectors: The Rubik’s cube of human gene therapy
Molecular Therapy, 2022 Defective genes account for ∼80% of the total of more than 7,000 diseases known to date. Gene therapy brings the promise of a one-time treatment option that will fix the errors in patient genetic coding.
A. Pupo +5 more
semanticscholar +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Objective To evaluate selumetinib exposure using therapeutic drug monitoring (TDM) in pediatric patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN), assess interpatient pharmacokinetic variability, and explore the relationship between drug exposure, clinical response, and adverse effects.
Janka Kovács +8 more
wiley +1 more source
GMP-manufactured CRISPR/Cas9 technology as an advantageous tool to support cancer immunotherapy
Journal of Experimental & Clinical Cancer ResearchBackground CRISPR/Cas9 system to treat human-related diseases has achieved significant results and, even if its potential application in cancer research is improving, the application of this approach in clinical practice is still a nascent technology ...
M Caforio +4 more
doaj +1 more source