Results 61 to 70 of about 8,605,496 (403)
Regulating Human Gene Therapy [PDF]
, 1985 Scientific developments have moved the public debate on genetic engineering to the issue of human gene therapy. Because so many important societal values must be weighed in deciding which, if any, of the first protocols for human gene therapy should be ...
Areen, Judith C.
core +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
Clinical Correlates of Anxiety and Depression After Diagnosis of a Pediatric Brain Tumor
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background The prevalence and clinical correlates of symptoms of anxiety and depression in pediatric patients with brain tumors are not well described. We aimed to identify clinical characteristics that are correlated with elevated symptoms of anxiety and depression following initial diagnosis.
Bryony J. Lucas +16 more
wiley +1 more source
Preoperative Cytopenia in Patients Affected by High‐Risk Neuroblastoma: Just a Matter of Platelets?
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background and Aims In patients affected by high‐risk neuroblastoma (HR‐NB), complete macroscopic resection (CMR) is associated with better outcomes. These patients are often cytopenic due to intensive induction regimens. The aim of the present study is to assess the impact of preoperative cytopenia on surgical outcome in patients with HR‐NB ...
Giorgio Persano +13 more
wiley +1 more source
Clinical and Biomedical Research, 2017 Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disorder due to deficiency of alpha-L-iduronidase (IDUA). Limitations such as need of weekly injection, high morbidity and mortality and high cost of the current treatments show the need for ...
Fabiano de Oliveira Poswar +5 more
doaj +2 more sources
Кардиоваскулярная терапия и профилактикаInformation on morbidity is presented in statistical reports for the entire population of multinational subjects of Russia, but population Biobanks contain information on individual peoples.Aim.
E. V. Balanovskaya +7 more
doaj +1 more source
Alcohol addiction: a molecular biology perspective. [PDF]
, 2015 Alcohol misuse represents worldwide an important risk factor for death and disability. Excessive alcohol consumption is widely diffused in different ethnicities and alcohol use is part of the lifestyle of both young and old people.
Ferraguti, Giampiero +2 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Adverse childhood experiences (ACEs) are stressful or traumatic events prior to age 18 that are known to have a lasting impact on individuals’ health and well‐being. There is a gap in understanding the relationship between ACEs and Other Life Stressors and health status for adults with sickle cell disease (SCD).
Marsha Treadwell +8 more
wiley +1 more source
Molecular Therapy: Nucleic Acids, 2016 Usher syndrome (USH) is the most common cause of combined deaf-blindness in man. The hearing loss can be partly compensated by providing patients with hearing aids or cochlear implants, but the loss of vision is currently untreatable.
Radulfus WN Slijkerman +15 more
doaj +1 more source
A new targeted CFTR mutation panel based on next-generation sequencing technology [PDF]
, 2017 Searching for mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR) is a key step in the diagnosis of and neonatal and carrier screening for cystic fibrosis (CF), and it has implications for prognosis and personalized therapy ...
Alberti, Luisella +10 more
core +2 more sources