freecell
ifreecell.bsky.social
freecell
@ifreecell.bsky.social
Discovery of Next Generation Therapeutics and Delivery Tools
Beyond Gene Editing: Exon Skipping Therapeutics
November 24, 2025 at 4:37 AM
Non-canonical miRNA functions
November 24, 2025 at 3:26 AM
Central role of skeletal muscle ageing in the pathogenesis of sarcopenia. www.sciencedirect.com/science/arti...

Potential pathways👇
✅insufficient myokine signalling (IL-6, 7, 15)
✅altered immune factors (pro-inflammatory profile)
✅impaired immune cell function
✅altered body composition
November 16, 2025 at 4:16 AM
NLRP3 inhibition by VTX3232 tempers inflammation resulting in reduced body weight, hyperglycemia, and hepatic steatosis in obese male mice. www.sciencedirect.com/science/arti...
November 16, 2025 at 4:09 AM
Advantages, applications and future directions of in vivo aptamer SELEX: a review dlvr.it/TLDzZN
Advantages, applications, and future directions of in vivo aptamer SELEX: A review
This review explores the advancements of in vivo SELEX in disease treatment, highlighting its ability to develop aptamers that overcome barriers such as the BBB and BRB. In vivo aptamers demonstrate p...
dlvr.it
November 12, 2025 at 4:59 AM
Two excellent reviews summarize recent progress with ADCs, highlighting the complexity of their design and immunological interactions, and offering insights to optimize therapeutic outcomes and guide next-generation development.
lnkd.in/eCxi8BeP
lnkd.in/eYeiAgeg
LinkedIn
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lnkd.in
November 11, 2025 at 12:02 PM
LNP for brain delivery
September 5, 2025 at 12:09 AM
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
August 18, 2025 at 2:23 AM
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
April 6, 2025 at 3:45 AM
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.
March 31, 2025 at 12:57 PM
Reducing off-target effects in siRNA: expert tips for better efficacy eclipsebio.com/eblogs/sirna...
March 29, 2025 at 10:51 PM
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.
March 29, 2025 at 4:55 PM
Research and Application of RNA Drugs in Kidney Diseases
March 22, 2025 at 2:16 AM
#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
March 8, 2025 at 7:09 PM
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
March 5, 2025 at 3:19 AM
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...
March 5, 2025 at 3:12 AM
The first step to use MS Exchange in an organization: servicedesk.msstate.edu/TDClient/45/...
March 3, 2025 at 2:14 AM
Mechanisms and comparison of ASOs. The ASOs regulate RNA function through different mechanisms. translationalneurodegeneration.biomedcentral.com/articles/10....
March 1, 2025 at 3:57 PM
www.mdpi.com/2076-3425/15... Modeling ALS with Patient-Derived iPSCs
March 1, 2025 at 3:46 PM
Functional assessment for therapeutic IgG mAbs academic.oup.com/abt/advance-...
March 1, 2025 at 3:40 PM
BBB delivery using extracellular vesicles? www.cell.com/molecular-th...
February 23, 2025 at 3:27 PM
Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications jbiomedsci.biomedcentral.com/articles/10....
February 23, 2025 at 3:23 PM
"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/
February 22, 2025 at 3:34 PM