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freecell @ifreecell.bsky.social · Sep 5

LNP for brain delivery

freecell @ifreecell.bsky.social · Aug 18

Kraus et al. report absolute miRNA concentrations required for productive cleavage of highly expressed transcripts and identify mouse miRNAs that reach this threshold in vivo.
bit.ly/44cYNvj

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freecell @ifreecell.bsky.social · Apr 6

Novel drug targets in 2024
www.nature.com/articles/d41...

Novel drug targets in 2024

Discover the world’s best science and medicine | Nature.com

www.nature.com

freecell @ifreecell.bsky.social · Mar 31

We developed a robust TDP-43-seeding platform for quantitative assessment of TDP-43 aggregate uptake, cell-to-cell spreading, and loss of function within living cells, while they progress toward pathology.

freecell @ifreecell.bsky.social · Mar 29

Reducing off-target effects in siRNA: expert tips for better efficacy eclipsebio.com/eblogs/sirna...

freecell @ifreecell.bsky.social · Mar 29

Understanding how the polyA tail interacts with L1 proteins, especially ORF2p, is critical for assessing these risks and developing strategies to enhance the safety of mRNA vaccines. We suggest conducting in vitro experiments to explore polyA tail modifications and their effects on L1 binding.

freecell @ifreecell.bsky.social · Mar 22

Research and Application of RNA Drugs in Kidney Diseases

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freecell @ifreecell.bsky.social · Mar 8

#PD: rare loss-of-function variants in ITSN1 increase Parkinson's disease risk by 10-fold—the largest effect size reported to date. This discovery provides new insights into the genetic architecture of PD and potential pathways for therapeutic development. www.cell.com/cell-reports...

Haploinsufficiency of ITSN1 is associated with a substantial increased risk of Parkinson’s disease

Spargo et al. analyzed whole-genome sequence data from ∼900,000 individuals and found that protein-truncating variants in ITSN1 confer a ∼10-fold increased risk of Parkinson’s disease. Functional stud...

www.cell.com

freecell @ifreecell.bsky.social · Mar 5

The Krainer laboratory uses multidisciplinary approaches to investigate the fundamental mechanisms and regulation of human pre-mRNA splicing, and the role of defective splicing in cancer and genetic diseases. www.krainerlab.cshl.edu/research

Krainer Lab - RESEARCH

OVERVIEW The Krainer laboratory uses multidisciplinary approaches to investigate the fundamental mechanisms and regulation of human pre-mRNA splicing, and the role of defective splicing in cancer and...

www.krainerlab.cshl.edu

freecell @ifreecell.bsky.social · Mar 5

GPCR drug discovery: new agents, targets and indications www.nature.com/articles/s41...

GPCR drug discovery: new agents, targets and indications - Nature Reviews Drug Discovery

G protein-coupled receptors (GPCRs) are one of the largest families of drug targets. This Review analyses drug discovery trends for GPCRs, covering compounds, targets and indications that have reached...

www.nature.com

freecell @ifreecell.bsky.social · Mar 5

This paper pushes me to rethink PCR—a routine assay that even a high school student can learn in a day. www.cell.com/molecular-th...

freecell @ifreecell.bsky.social · Mar 3

The first step to use MS Exchange in an organization: servicedesk.msstate.edu/TDClient/45/...

freecell @ifreecell.bsky.social · Mar 1

Mechanisms and comparison of ASOs. The ASOs regulate RNA function through different mechanisms. translationalneurodegeneration.biomedcentral.com/articles/10....

freecell @ifreecell.bsky.social · Mar 1

www.mdpi.com/2076-3425/15... Modeling ALS with Patient-Derived iPSCs

freecell @ifreecell.bsky.social · Mar 1

Functional assessment for therapeutic IgG mAbs academic.oup.com/abt/advance-...

freecell @ifreecell.bsky.social · Feb 23

BBB delivery using extracellular vesicles? www.cell.com/molecular-th...

freecell @ifreecell.bsky.social · Feb 23

Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications jbiomedsci.biomedcentral.com/articles/10....

freecell @ifreecell.bsky.social · Feb 22

"The versatility of CRAAVR cells for the streamlined testing and batch validation of AAVs for research and therapeutic applications."----but cannot be applied to all serotypes, such as AAV4. pubmed.ncbi.nlm.nih.gov/39944269/