Splicing News and Research

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Exonate first-in-class eye drop Phase Ib/IIa trial data demonstrate safety and biological activity in treatment of diabetic retinopathy and diabetic macular oedema

EXN407 is the first topical SRPK1 inhibitor to demonstrate both safety and signals of biological response as monotherapy for these indications.

5 Mar 2024

Research identifies genetic change associated with human tail loss

A genetic change in our ancient ancestors may partly explain why humans don't have tails like monkeys, finds a new study led by researchers at NYU Grossman School of Medicine.

28 Feb 2024

Mapping the brain: How cell types and location influence Parkinson’s disease

A single-cell spatial transcriptome analysis on murine brain expression in age and disease using a Parkinson's disease.

27 Feb 2024

Novel splicing mechanism for short introns discovered

Researchers confirm that the established pre-mRNA splicing mechanism that appears in textbooks cannot work in a subset of human short introns: A novel SAP30BP–RBM17 complex-dependent splicing has been uncovered.

15 Feb 2024

Decoding the differences between two vital RNA transport complexes

Genetic expression, often leading to protein synthesis, requires a complex coordination of molecular machinery across several stages.

8 Feb 2024

Researchers discover mechanism that protects tissue after faulty gene expression

The genetic material, in the form of DNA, contains the information that is crucial for the correct functioning of every human and animal cell.

2 Feb 2024

New statistical tool improves the ability to find genetic variants that cause disease

A new statistical tool developed by researchers at the University of Chicago improves the ability to find genetic variants that cause disease.

26 Jan 2024

New workflow identifies shared cancer targets, advancing immunotherapy

Researchers developed the Splicing Neo Antigen Finder (SNAF) workflow, integrating deep learning to identify shared immunogenic neoantigens in cancers, offering new targets for cancer immunotherapy, particularly in melanoma and ovarian cancer.

23 Jan 2024

Innovative computational tool unearths hidden cancer targets for immunotherapy

An innovative computational tool dubbed "SNAF" may help the research world bring the emerging promise of cancer immunotherapy to a wider range of patients, according to a study published Jan. 17, 2024, in Science Translational Medicine.

17 Jan 2024

Natural PLA2 inhibitor from marine fungus combats fatty liver disease by silencing lipotoxic pathways

This new article publication from Acta Pharmaceutica Sinica B, discusses the identification of a natural PLA2 inhibitor from the marine fungus Aspergillus sp. c1 for MAFLD treatment that suppressed lipotoxicity by inhibiting the IRE-1α/XBP-1s axis and JNK signaling.

16 Jan 2024

RBM5 identified as key regulator of HOXA9 in acute myeloid leukemia

The protein HOXA9 is overexpressed in most acute myeloid leukemia (AML) cases and is associated with poor patient outcomes.

12 Jan 2024

Revolutionary RNA-based switch offers new control over gene expression in mammalian cells

Researchers developed an RNA-based switch, the pA regulator system, to control gene expression in mammalian cells by modulating synthetic polyA signal cleavage, offering a novel approach for gene therapy applications.

4 Jan 2024

Calcium channel blockers reverse muscle weakness in animal model of myotonic dystrophy type 1

New research has identified the specific biological mechanism behind the muscle dysfunction found in myotonic dystrophy type 1 (DM1) and further shows that calcium channel blockers can reverse these symptoms in animal models of the disease.

2 Jan 2024

From Neanderthals to now: Tracing the evolution of human sleep cycles

Researchers examined the differences in circadian biology between the ancestors of modern humans and the archaic hominins such as Denisovans and Neanderthals and the contribution of archaic introgression in human chronotypes by using genomes to trace chronotype evolution from archaic hominins to modern-day humans.

19 Dec 2023

Study could pave the way for synergistic therapeutic combinations to better treat SF3B1-mutant MDS

A new study from researchers with Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine and collaborating organizations provides insight into the underlying mechanisms of gene mutations commonly seen in patients with myelodysplastic syndromes (MDS) and other myeloid neoplasms.

11 Dec 2023

Splicing News and Research

Exonate first-in-class eye drop Phase Ib/IIa trial data demonstrate safety and biological activity in treatment of diabetic retinopathy and diabetic macular oedema

Research identifies genetic change associated with human tail loss

Mapping the brain: How cell types and location influence Parkinson’s disease

Novel splicing mechanism for short introns discovered

Decoding the differences between two vital RNA transport complexes

Researchers discover mechanism that protects tissue after faulty gene expression

New statistical tool improves the ability to find genetic variants that cause disease

New workflow identifies shared cancer targets, advancing immunotherapy

Innovative computational tool unearths hidden cancer targets for immunotherapy

Natural PLA2 inhibitor from marine fungus combats fatty liver disease by silencing lipotoxic pathways

RBM5 identified as key regulator of HOXA9 in acute myeloid leukemia

Revolutionary RNA-based switch offers new control over gene expression in mammalian cells

Calcium channel blockers reverse muscle weakness in animal model of myotonic dystrophy type 1

From Neanderthals to now: Tracing the evolution of human sleep cycles

Study could pave the way for synergistic therapeutic combinations to better treat SF3B1-mutant MDS

Arxspan BioDrive: A Versatile Solution for Molecular Biology

New insights into the interplay between prions and TDP-43 in neurodegenerative pathology

Study identifies how three novel genes cause neurodevelopmental disorders

Exonate announces successful completion of Phase Ib/IIa trial in diabetic macular oedema

Gene splicing determines CD20 levels and response to monoclonal antibody therapy

Advancing Intracellular Delivery with LumiPore®

How ELRIG Is Helping Mid-Career Scientists Navigate Leadership, Career Change and Redundancy

The role of UV-Vis in biopharma QC

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