Results 171 to 180 of about 8,605,496 (403)
Adaptaquin selectively kills glioma stem cells while sparing differentiated brain cells. Transcriptomic and proteomic analyses show Adaptaquin disrupts iron and cholesterol homeostasis, with iron chelation amplifying cytotoxicity via cholesterol depletion, mitochondrial dysfunction, and elevated reactive oxygen species.
Adrien M. Vaquié +16 more
wiley +1 more source
Central nervous system (CNS) disorders are driven by complex genetic and epigenetic factors. While gene‐based interventions (siRNA, mRNA, CRISPR systems, etc.) hold transformative potential, their clinical application is severely constrained by ...
Fuming Liang +4 more
doaj +1 more source
Extravascular factor IX after gene therapy in hemophilia B, does it matter?
Gene therapy will very likely change the treatment paradigm of hemophilia B in the coming years. For the majority of patients, adjunctive exogenous factor (F)IX clotting factor concentrate will continue to be needed in case of surgery or bleeding ...
Quentin Van Thillo, Cédric Hermans
doaj +1 more source
The Cone Dysfunction Syndromes [PDF]
The cone dysfunction syndromes are a heterogeneous group of inherited, predominantly stationary retinal disorders characterised by reduced central vision, and varying degrees of colour vision abnormalities, nystagmus and photophobia.
Aboshiha, J +4 more
core
A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics
James W. Hawkins
openalex +1 more source
Therapy-related Acute Lymphoblastic Leukaemia has a Unique Genetic Profile Compared to De Novo Acute Lymphoblastic Leukaemia [PDF]
Hye Won Kook +7 more
openalex +1 more source
This study integrates transcriptomic profiling of matched tumor and healthy tissues from 32 colorectal cancer patients with functional validation in patient‐derived organoids, revealing dysregulated metabolic programs driven by overexpressed xCT (SLC7A11) and SLC3A2, identifying an oncogenic cystine/glutamate transporter signature linked to ...
Marco Strecker +16 more
wiley +1 more source
Chronic lower limb ischemia is a debilitating condition, particularly prevalent among elderly patients and individuals ineligible for revascularization procedures.
Igor V. Samatoshenkov +8 more
doaj +1 more source
How Can We Move Clinical Genomics Beyond the Hype? [PDF]
Examines the debate over increased use of genetic testing, due in part to lax regulation, and its consequences: wasteful spending, patient harm, and health system challenges.
Michael L. Millenson
core
This study characterizes the responses of primary acute myeloid leukemia (AML) patient samples to the MCL‐1 inhibitor MIK665. The results revealed that monocytic differentiation is associated with MIK665 sensitivity. Conversely, elevated ABCB1 expression is a potential biomarker of resistance to the treatment, which can be overcome by the combination ...
Joseph Saad +17 more
wiley +1 more source

