Results 221 to 230 of about 4,021 (283)

Dual IRE1 targets: Determinants of the cell fate?

open access: yesThe FEBS Journal, EarlyView.
IRE1 is an ER stress sensor that restores protein homeostasis through two signaling activities: XBP1s, which upregulates its target gene expression or RIDD which downregulates its target transcripts. We recently identified Dual IRE1 Targets (DIT) which are modulated by both XBP1s and RIDD activities.
Eva Billat   +3 more
wiley   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

open access: yesHaemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach   +2 more
wiley   +1 more source

The Corrective Role of Melatonin in Synergism of Dark Deprivation and CCl4 Intoxication in the Pathogenesis of Liver Damage a in Rats. [PDF]

open access: yesCurr Issues Mol Biol
Grabeklis SA   +6 more
europepmc   +1 more source

Gene Editing for Haemophilia—The Next Frontier

open access: yesHaemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti   +3 more
wiley   +1 more source

Erythropoietic protoporphyrias: Pathogenesis, diagnosis and management. [PDF]

open access: yesLiver Int
Minder AE   +4 more
europepmc   +1 more source

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