SCI Abstract

search
+
AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system
AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system
Adeno-associated virus serotype 2 (AAV2) remains one of the most common vectors for CNS gene delivery. Yet, its long-term ...
Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease
Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease
The benefit of early diagnosis and treatment has been demonstrated in animal models of several lysosomal storage diseases....
A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy
A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy
Charcot-Marie-Tooth disease type 4C is a demyelinating neuropathy caused by loss of function mutations in the SH3TC2 gene,...
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
Hemophilia A (HA), an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency, is primarily managed with exoge...
Research progress and development strategies of antibody-oligonucleotide conjugates
Research progress and development strategies of antibody-oligonucleotide conjugates
Antibody-oligonucleotide conjugates (AOCs) effectively integrate the delivery capability of antibodies with the specific g...
Characterization of CAR-T cell factors that contribute to myeloid cell activation
Characterization of CAR-T cell factors that contribute to myeloid cell activation
Chimeric antigen receptor (CAR) T cell therapies have shown remarkable success in the treatment of hematologic cancers; ho...
DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation
DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation
The inhibition of estrogen receptor (ER)-mediated genomic signaling in ER-positive cancer cells has long been a primary fo...
Environmental and developmental factors shape anti-AAV immunity in pigs
Environmental and developmental factors shape anti-AAV immunity in pigs
Use of adeno-associated virus (AAV) vectors has revolutionized in vivo gene therapy, but the presence of pre-existing neut...
Possible regulatory network and associated pathways governing the expression of in
Possible regulatory network and associated pathways governing the expression of in
With the day to day increase in energy consumption due to increase in urbanization production of bioethanol is highly in d...
Tolerance of the non-conventional yeast BT3 to cadmium exposure: a genomic characterization
Tolerance of the non-conventional yeast BT3 to cadmium exposure: a genomic characterization
Cadmium, a significant environmental heavy metal contaminant, poses considerable threats to human health. Cadmium detoxifi...
Genomic characterization and analysis of isolate 1-Jan, a novel pathogenic strain infecting
Genomic characterization and analysis of isolate 1-Jan, a novel pathogenic strain infecting
Fusarium species are pathogens affecting the broodstock of farm-raised Fannabin prawns, resulting in significant economic ...
Epigenetic regulation of pathogenicity in fungi
Epigenetic regulation of pathogenicity in fungi
Histone modifications regulate vital pathogenic features necessary for opportunistic fungi to cause disease. More specific...
Genomic surveillance of vancomycin-resistant : a study on Resistome, Plasmidome, and mobilome profiling
Genomic surveillance of vancomycin-resistant : a study on Resistome, Plasmidome, and mobilome profiling
Vancomycin-resistant enterococci (VRE) are critical nosocomial pathogens, classified as high priority by the World Health ...
Genome characterization of species from the rice rhizosphere: a potential plant growth promoting rhizobacteria (PGPR)
Genome characterization of species from the rice rhizosphere: a potential plant growth promoting rhizobacteria (PGPR)
Plant-microbe interactions in the rice rhizosphere play a pivotal role for crop productivity. Acinetobacter spp. are known...
The CRISPR-cas repertoire of : insights from genomic data
The CRISPR-cas repertoire of : insights from genomic data
The genus Kluyvera has gained increasing attention due to their emerging role as opportunistic pathogens and their antibio...
Is heptelidic (koningic) acid a microbial hormone that regulates secondary metabolism in the biocontrol fungus ?
Is heptelidic (koningic) acid a microbial hormone that regulates secondary metabolism in the biocontrol fungus ?
Trichoderma virens, a plant- beneficial fungus, has the ability to produce volatile and non-volatile secondary metabolites...
Loss of alters metabolome in response to MCHM, a synthetic hydrotrope
Loss of alters metabolome in response to MCHM, a synthetic hydrotrope
4-methylcyclohexane methanol (MCHM), an alicyclic alcohol, is a synthetic hydrotrope that induces a starvation response by...
Spermidine synthase is essential for the trophic growth, stress response, and pathogenicity of
Spermidine synthase is essential for the trophic growth, stress response, and pathogenicity of
Spermidine synthase (SPDS) is a key enzyme in polyamine biosynthesis, a metabolic pathway that plays a critical regulatory...
When the genome learned its own vocabulary
In this Journal Club article, Maria Brbić discusses DeepSEA, a pioneering deep convolutional neural network to predic...
Mechanisms of DNA specificity in chromatin remodelling
In this Journal Club, Lu Bai highlights a 2021 paper by Donovan et al. that brought mechanistic clarity to the question of...
The epigenetic nature of X chromosome inactivation
In this Journal Club, Claudia Keller Valsecchi recalls two seminal studies by Csankovszki et al. and by Wutz and Jaenisch ...
Nutrition signals the brain to reactivate quiescent neural stem cells
In this Journal Club article, Hongyan Wang highlights two papers that elucidated the signalling mechanisms and inter-organ...
Prime editing: redefining precision genome editing
In this Journal Club article, Qiupeng Lin recalls the 2019 paper by Anzalone et al. that introduced prime editing, and ref...
RNA design across eras: from covariance models to modern generative AI
In this Journal Club, Annalisa Marsico describes a 1994 paper by Eddy and Durbin that formalized RNA sequence analysis wit...
Resolving structural variants in pangenome graphs with Swave
Resolving structural variants in pangenome graphs with Swave
In this Tools of the Trade article, Songbo Wang introduces Swave, an AI-enhanced sequence-to-image method capable of detec...
Evolutionary genetics of ageing
Evolutionary genetics of ageing
Modern humans now routinely survive to advanced ages, in far greater proportions than ancestral populations, and thus expe...
Biallelic WDR91 variants cause a neurodevelopmental disorder through impaired endosomal maturation and autophagy dysregulation
Biallelic WDR91 variants cause a neurodevelopmental disorder through impaired endosomal maturation and autophagy dysregulation
medRxiv - the preprint server for biology, operated by openRxiv, a nonprofit organization dedicated to advancing scientifi...
A complex duplication overlapping FBRSL1 implicated in a developmental and epileptic encephalopathy
A complex duplication overlapping FBRSL1 implicated in a developmental and epileptic encephalopathy
medRxiv - the preprint server for biology, operated by openRxiv, a nonprofit organization dedicated to advancing scientifi...
Genomic ascertainment of PALB2-related cancer predisposition
Genomic ascertainment of PALB2-related cancer predisposition
medRxiv - the preprint server for biology, operated by openRxiv, a nonprofit organization dedicated to advancing scientifi...
RD-Embed: Unified representations of rare-disease knowledge from clinical records
RD-Embed: Unified representations of rare-disease knowledge from clinical records
medRxiv - the preprint server for biology, operated by openRxiv, a nonprofit organization dedicated to advancing scientifi...
Load More
format_indent_increase