Results 111 to 120 of about 8,660,824 (403)

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, Lucia Susani, Alessandra Castelli, Teruhiko Suzuki, Takahiko Hara, Letizia Straniero, Stefano Duga, Dario Strina, Stefano Mantero, Elena Caldana, Lucia Sergi Sergi, Anna Villa, Paolo Vezzoni   +12 more
doaj   +1 more source

Genetic screening in new onset inflammatory bowel disease during anti-interleukin 17 therapy: unmet needs and call for action [PDF]

, 2021
Carlo Alberto Maronese, Enrico Zelin, Chiara Moltrasio, Giovanni Genovese, Angelo Valerio Marzano   +4 more
openalex   +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

Enteropathogenic E. coli shows delayed attachment and host response in human jejunum organoid‐derived monolayers compared to HeLa cells

FEBS Letters, EarlyView.
Enteropathogenic E. coli (EPEC) infects the human intestinal epithelium, resulting in severe illness and diarrhoea. In this study, we compared the infection of cancer‐derived cell lines with human organoid‐derived models of the small intestine. We observed a delayed in attachment, inflammation and cell death on primary cells, indicating that host ...
Mastura Neyazi, Pilar Samperio Ventayol, Natalie Burkard, Nicolas Schlegel, Carmen Aguilar, Sina Bartfeld   +5 more
wiley   +1 more source

Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review

Gene Therapy, 2021
N. M. Al-Namnam, S. N. Jayash, F. Hariri, Z. Rahman, M. Alshawsh   +4 more
semanticscholar   +1 more source

Polymorphism in glutathione S-transferase P1 is associated with susceptibility to chemotherapyinduced leukemia [PDF]

, 2001
Glutathione S-transferases (GSTs) detoxify potentially mutagenic and toxic DNA-reactive electrophiles, including metabolites of several chemotherapeutic agents, some of which are suspected human carcinogens.
Allan, J.M., Wild, C.P., Rollinson, S., Willett, E.V., Moorman, A.V., Dovey, G.J., Roddam, P.L., Roman, E., Cartwright, R.A., Morgan, G.J.   +9 more
core   +4 more sources

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source

Expanding the Phenotype of CYFIP2‐Related Developmental Epileptic Encephalopathy: Case Report and Literature Review

Annals of the Child Neurology Society
Background Pathogenic CYFIP2 variants cause developmental and epileptic encephalopathy (DEE), characterized by early‐onset intractable epilepsy and developmental delay. The disease course has not been delineated.
Michaela Squire, Jenna K. Lea, Zheng (Jane) Fan, Senyene E. Hunter   +3 more
doaj   +1 more source

LDLR-Gene therapy for familial hypercholesterolaemia: Problems, progress, and perspectives [PDF]

, 2010
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates.
Al-Allaf, FA, Coutelle, C, David, A, Harbottle, R, Themis, M, Waddington, SN   +5 more
core   +3 more sources

Reciprocal control of viral infection and phosphoinositide dynamics

FEBS Letters, EarlyView.
Phosphoinositides, although scarce, regulate key cellular processes, including membrane dynamics and signaling. Viruses exploit these lipids to support their entry, replication, assembly, and egress. The central role of phosphoinositides in infection highlights phosphoinositide metabolism as a promising antiviral target.
Marie Déborah Bancilhon, Bruno Mesmin
wiley   +1 more source