Results 71 to 80 of about 5,467,287 (313)
Splicing modulation therapy in the treatment of genetic diseases
, 2014 Virginia Arechavala-Gomeza,1 Bernard Khoo,2 Annemieke Aartsma-Rus3 1Neuromuscular Disorders Group, BioCruces Health Research Institute, Barakaldo, Bizkaia, Spain; 2Endocrinology, Division of Medicine, University College London, London, UK; 3Department ...
Khoo B +2 more
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Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Young adult childhood cancer survivors (YA‐CCS) have an increased risk of treatment‐related health complications throughout their lives. Lifelong survivorship care is recommended, yet many YA‐CCS experience disruptions in care during pediatric‐to‐adult transitions and do not receive recommended survivorship care.
Kevin Shanchen Pu +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
Identifying Patterns of Hospitalizations Among Childhood Cancer Survivors: A Population‐Based Study
Pediatric Blood &Cancer, EarlyView.ABSTRACT Background Childhood cancer survivors (CCSs) experience more hospitalizations than the general population. We aimed to identify distinct hospitalization patterns among Queensland CCSs and the associated characteristics. Methods Cases (aged ≤14 years) diagnosed between 1995 and 2019 while residents of Queensland were identified through the ...
Hai Pham +7 more
wiley +1 more source
Molecular Therapy: Nucleic Acids, 2012 Leber congenital amaurosis (LCA) is the most severe form of inherited retinal degeneration, with an onset in the first year of life. The most frequent mutation that causes LCA, present in at least 10% of individuals with LCA from North-American and ...
Rob WJ Collin +5 more
doaj +1 more source
Genetic therapies for neurological diseases
Pharmacological ReviewsOften, gene therapy reviews concentrate upon specific therapeutic modalities-particularly either viral vector-mediated or a nonviral approach. Here, we draw together a comprehensive array of knowledge across the field of genetic therapy for genetic neurological disease.
Ahad A. Rahim +7 more
openaire +2 more sources
Pediatric Blood &Cancer, EarlyView.ABSTRACT Pediatric refractory immune thrombocytopenia (rITP) is generally characterized by persistent thrombocytopenia and failure to achieve a sustained response to multiple standard therapies. We report a 4‐year‐old female with congenital tufting enteropathy, post‐intestinal transplant patient on immunosuppressants, who developed severe rITP ...
Sarah Hsieh +4 more
wiley +1 more source
Targeted genetic repair: an emerging approach to genetic therapy [PDF]
Journal of Clinical Investigation, 2003 The last two decades have heralded a remarkable increase in our understanding of the genetic basis of disease and the endogenous mechanisms responsible for the repair of genomic DNA and the processing of RNA. Targeted gene repair is a powerful yet controversial technique developed to direct base changes in chromosomal genes, while RNA repair is an ...
openaire +2 more sources
Journal of Proton Therapy: Call for Papers
, 2015 Journal of Proton Therapy (JPT) is an international open access, peer-reviewed journal, which publishes original research, technical reports, reviews, case reports, editorials, and other materials on proton therapy with focus on radiation oncology ...
Proton Therapy, Journal of
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, 2020 "Created: 3/16/2020..."--Document properties ...
Ohio Occupational Therapy, Physical Therapy, and Athletic Trainers Board
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