Results 61 to 70 of about 11,459,182 (339)
Entering the Modern Era of Gene Therapy.
Annual Review of Medicine, 2019 Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase.
X. Anguela, K. High
semanticscholar +1 more source
Evolutionary interplay between viruses and R‐loops
FEBS Letters, EarlyView.Viruses interact with specialized nucleic acid structures called R‐loops to influence host transcription, epigenetic states, latency, and immune evasion. This Perspective examines the roles of R‐loops in viral replication, integration, and silencing, and how viruses co‐opt or avoid these structures.
Zsolt Karányi +4 more
wiley +1 more source
Autoimmune dysregulation and purine metabolism in adenosine deaminase (ADA)-deficiency
Frontiers in Immunology, 2012 Genetic defects in the adenosine deaminase (ADA) gene are among the most common causes for severe combined immunodeficiency (SCID). ADA-SCID patients suffer from lymphopenia, severely impaired cellular and humoral immunity, failure to thrive and ...
Aisha Vanessa Sauer +5 more
doaj +1 more source
BMC Genomics, 2020 An amendment to this paper has been published and can be accessed via the original article.
Camilo Breton +4 more
doaj +1 more source
Disruption of SETD3‐mediated histidine‐73 methylation by the BWCFF‐associated β‐actin G74S mutation
FEBS Letters, EarlyView.The β‐actin G74S mutation causes altered interaction of actin with SETD3, reducing histidine‐73 methylation efficiency and forming two distinct actin variants. The variable ratio of these variants across cell types and developmental stages contributes to tissue‐specific phenotypical changes. This imbalance may impair actin dynamics and mechanosensitive
Anja Marquardt +8 more
wiley +1 more source
Oral Diseases, 2013 Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire +2 more sources
Gene Therapy for Hemophilia [PDF]
Molecular Therapy, 2016 The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [FVIII] for hemophilia A and factor IX [FIX] for hemophilia B).
Nienhuis, AW, Nathwani, AC, Davidoff, AM
openaire +4 more sources
Early induction of cytokine release syndrome by rapidly generated CAR T cells in preclinical models
EMBO Molecular MedicineCytokine release syndrome (CRS) is a significant side-effect of conventional chimeric antigen receptor (CAR) T-cell therapy. To facilitate patient accessibility, short-term (st) CAR T cells, which are administered to patients only 24 h after vector ...
Arezoo Jamali +5 more
doaj +1 more source
Circulating histones as clinical biomarkers in critically ill conditions
FEBS Letters, EarlyView.Circulating histones are emerging as promising biomarkers in critical illness due to their diagnostic, prognostic, and therapeutic potential. Detection methods such as ELISA and mass spectrometry provide reliable approaches for quantifying histone levels in plasma samples.
José Luis García‐Gimenez +17 more
wiley +1 more source
FEBS Letters, EarlyView.This study reveals how prime editing guide RNA (pegRNA) secondary structure and reverse transcriptase template length affect prime editing efficiency in correcting the phospholamban R14del cardiomyopathy‐associated mutation. Insights support the design of structurally optimized enhanced pegRNAs for precise gene therapy.
Bing Yao +7 more
wiley +1 more source