Results 51 to 60 of about 5,467,287 (313)
Gene therapy for pediatric genetic kidney diseases
, 2023 Genetic kidney disease is the main cause of chronic kidney disease in children. While the pathogenic genes associated with most genetic kidney diseases have been identified, the underlying mechanisms of disease initiation remain ambiguous, and effective ...
Lirong Zhang +4 more
core +1 more source
Applying the Ecological Model of Human Performance and the SlutWalk Movement to Support those Affected by Rape Culture in the Context of Occupational Therapy [PDF]
, 2023 Improving the occupational lives of others is at the heart of occupational therapy practice. Advocacy for clientele is central to this improvement, both at individual and structural levels.
Klukken, Angela +3 more
core +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Numerous international studies have reported declines in new cancer diagnoses, delayed diagnoses and disruptions in cancer treatment following the implementation of COVID‐19 pandemic public health measures, raising concerns that these effects may ultimately contribute to increased cancer mortality.
Friederike Erdmann +8 more
wiley +1 more source
GENE THERAPY IN MUCOPOLYSACCHARIDOSIS TYPE IIIA: CASE REPORTS
Slovenska pediatrija, 2022 Mucopolysaccharidoses are a group of rare lysosomal storage diseases. The clinical signs develop gradually, the impairment is progressive and multiple organs are affected. With the currently known treatment options, the patient cannot be cured.
Benjamin Lah +7 more
doaj +1 more source
A theological consideration of ethical issues raised by human genetic manipulation, with particular reference to gene therapy [PDF]
, 1999 Human genetic manipulation is considered in terms of genetic testing and screening, gene therapy and enhancement, and reproductive cloning. Deontological and utilitarian approaches to ethical decision-making prove less than satisfactory.
Elkington, Audrey Anne
core
Genetic and Genomic Landscape of Secondary and Therapy-Related Acute Myeloid Leukemia
, 2020 A subset of acute myeloid leukemia (AML) arises either from an antecedent myeloid malignancy (secondary AML, sAML) or as a complication of DNA-damaging therapy for other cancers (therapy-related myeloid neoplasm, t-MN).
Alexandra Higgins, Mithun Vinod Shah
core +1 more source
Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders [PDF]
, 2022 We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration ...
Tauscher-Wisniewski, Sitra +16 more
core +1 more source
Referral Patterns and Diagnostic Timeliness in Pediatric Cancer: A Hospital‐Based Study in Indonesia
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Timely diagnosis and treatment are critical for improving survival among children with cancer. In low‐ and middle‐income countries (LMICs), delays are common and may be influenced by fragmented referral pathways and diagnostic limitations.
Nur Melani Sari +5 more
wiley +1 more source
Chromosome Transplantation: A Possible Approach to Treat Human X-linked Disorders
Molecular Therapy: Methods & Clinical Development, 2020 Many human genetic diseases are associated with gross mutations such as aneuploidies, deletions, duplications, or inversions. For these “structural” disorders, conventional gene therapy, based on viral vectors and/or on programmable nuclease-mediated ...
Marianna Paulis +12 more
doaj +1 more source
Pediatric Blood &Cancer, EarlyView.ABSTRACT Relapsed and/or refractory (R/R) pediatric acute leukemia carries a dismal prognosis, largely driven by chemoresistance to conventional salvage therapy. The BH3‐mimetic venetoclax, combined with chemotherapy or hypomethylating agents, has demonstrated efficacy in small clinical trials.
Katherine S. Colman +9 more
wiley +1 more source