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Biomarkers

Study identifies CIAO1 as a neuromuscular disease gene

March 6, 2024
Iron-sulfur clusters are co-factors that are involved in several biological processes, such as oxidative phosphorylation, enzymatic reactions, and DNA replication and repair. Scientists from the National Institutes of Health (NIH) have presented a study regarding the clinical manifestations associated with a novel neuromuscular disease gene – CIAO1 – which encodes probable cytosolic iron-sulfur protein assembly protein CIAO1 and is an essential member in the cytoplasmic iron-sulfur assembly machinery.
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Virus particles attacking neuron
HIV/AIDS

CROI 2024: HIV is active in the CNS despite ART

March 5, 2024
By Mar de Miguel
On March 4, 2024, several groups of scientists discussed the challenges of investigating the effects of HIV in the central nervous system (CNS) at the oral abstract session on neuropathogenesis of HIV held during the 31st Conference on Retroviruses and Opportunistic Infections (CROI), in Denver. A cure for HIV will require eliminating the virus in all its reservoirs, those tissues where HIV remains latent but retains the capacity for reactivation and replication. However, despite antiretroviral therapy (ART), the virus could continue to replicate continuously at a low level in some reservoirs, including the CNS.
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Doctor examining child in wheelchair
Neurology/Psychiatric

New murine model resembles human phenotype for Becker muscular dystrophy

March 5, 2024
A male patient who harbored a deletion in the intron 7-exon 8 region of the dystrophin gene, DMD, was showing symptoms that matched with Becker muscular dystrophy.
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Brain
Neurology/Psychiatric

Brain-penetrant TYK2 inhibitor shows efficacy in models of neuroinflammation

March 5, 2024
Researchers from Alumis Inc. have presented preclinical data for a novel, potential first-in-class tyrosine kinase 2 (TYK2) inhibitor – A-005.
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Multiple sclerosis
Neurology/Psychiatric

Find Therapeutics reports preclinical pharmacology of new remyelinating agent

March 4, 2024
Semaphorin 3A signaling, through the plexin A1/neuropilin 1 (PLXA1/NRP1) receptor complex, is known to disrupt the differentiation and migration of oligodendrocyte precursor cells and mature remyelinating oligodendrocytes. Both semaphorin 3A and plexin A1 are up-regulated in the central nervous system of patients with multiple sclerosis.
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Illustration of DNA, digestive system
Biomarkers

miR-21, miR-135b as biomarkers of IBD-related colorectal cancer

March 1, 2024
Researchers from University of Padova have presented data from a study that aimed to investigate the role of microRNAs (miRs) in the development and progression of inflammatory bowel disease (IBD)-related colorectal cancer (CRC).
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Cancer

GT-19630 inhibits proliferation, promotes apoptosis and blocks migration of breast cancer cells

March 1, 2024
At this week’s EACR-AACR-ICAR meeting in Dublin, Suzhou Kintor Pharmaceuticals Inc. presented preclinical data for the novel MYC- and GSPT1-targeting molecular glue candidate, GT-19630, being developed for the treatment of breast cancer.
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Immuno-oncology

Preclinical data support ongoing clinical development of MAb and ADC for CLDN18.2+ tumors

March 1, 2024
Tumors in the stomach and pancreas are known to express claudin 18.2 (CLDN18.2) and the absence of expression in normal tissues makes it an attractive therapeutic target.
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Cancer

Second-gen RNA pol I inhibitor has robust, specific activity

Feb. 29, 2024
The transcription of ribosomal RNA by RNA Polymerase I (Pol I) is an essential step for cells to grow and thus is considered a therapeutic target for cancer treatment.
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Illustration of ovary, uterus and endometrial cancer.
Immuno-oncology

CLDN6-23-ADC shows promise for ovarian, endometrial tumors

Feb. 29, 2024
Claudin-6 (CLDN6) is highly expressed in several cancer types, including ovarian and endometrial cancers, while very low or no expression is found in normal tissues.
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