freecell

LNP for brain delivery

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

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

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.

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

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.

Research and Application of RNA Drugs in Kidney Diseases

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...

#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...

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...

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

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...

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

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...

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

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

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

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

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

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

"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/

Gut-brain pathways and sites of action of endogenous and exogenous GLP-1 signaling academic.oup.com/endo/article...

Three sources of myofibroblasts pmc.ncbi.nlm.nih.gov/articles/PMC...

library preparation protocols used to study distinct RNA species

Keystone: Drug Delivery to the Brain www.keystonesymposia.org/conferences/...

Choose a right control for immunofluorescence assay

massively parallel in vivo Perturb-seq screening www.nature.com/articles/s41...

Improved workflow for Guide-Seq www.biorxiv.org/content/10.1...

Therapeutic PCSK9 targeting: Inside versus outside the hepatocyte? pubmed.ncbi.nlm.nih.gov/39947256/

Protocol overview for single-cell CRISPR screening in mouse brain.

Fc Gamma Receptor Polymorphisms in Antibody Therapy: Implications for Bioassay Development to Enhance Product Quality academic.oup.com/abt/advance-...

Protein-Based Degraders: From Chemical Biology Tools to Neo-Therapeutics The nascent field of targeted protein degradation (TPD) could revolutionize biomedicine due to the ability of degrader molecules to selectively modulate disease-relevant proteins. A key limitation to the broad application of TPD is its dependence on small-molecule ligands to target proteins of interest. This leaves unstructured proteins or those lacking defined cavities for small-molecule binding out of the scope of many TPD technologies. The use of proteins, peptides, and nucleic acids (otherwise known as “biologics”) as the protein-targeting moieties in degraders addresses this limitation. In the following sections, we provide a comprehensive and critical review of studies that have used proteins and peptides to mediate the degradation and hence the functional control of otherwise challenging disease-relevant protein targets. We describe existing platforms for protein/peptide-based ligand identification and the drug delivery systems that might be exploited for the delivery of biologic-based degraders. Throughout the Review, we underscore the successes, challenges, and opportunities of using protein-based degraders as chemical biology tools to spur discoveries, elucidate mechanisms, and act as a new therapeutic modality.

Great overview from the team of Laura Dassama at Stanford on the state of the art protein-based degraders used for TPD!
pubs.acs.org/doi/10.1021/...

Gains in early-onset dementia with progranulin open new paths for drug discovery - Nature Biotechnology The first clinical results in patients with a genetic form of frontotemporal dementia (FTD) show that enhancing progranulin in the brain may halt disease progression. If successful, this potentially d...

The first clinical results in patients with a genetic form of FTD show that enhancing progranulin in the brain may halt disease progression. If successful, this disease-modifying approach may uncover new avenues for treating other neurodegenerative diseases. www.nature.com/articles/s41...