ICAHN School of Medicine At Mount Sinai

ICAHN School of Medicine At Mount Sinai logo
🇺🇸United States
Ownership
Private, Subsidiary
Established
1963-01-01
Employees
5K
Market Cap
-
Website
http://www.mssm.edu
nature.com
·

Abstracts from the 57th European Society of Human Genetics (ESHG) Conference

C22.1: KS1 iPSCs showed lower functional KMT2D expression, H3K4me1/2 levels, and differential gene expression related to KS1 phenotypes. C22.2: Single-cell genomics identified cell-type variation in expression and chromatin, regulatory elements, and eQTLs, facilitating precision medicine for neuropsychiatric disorders. C22.3: XDP-TE in TAF1 gene linked to XDP; ZNF91 establishes H3K9me3 and DNA methylation, crucial for TAF1 regulation. C22.4: MORC2 mutations cause diverse neurological disorders; multi-omics analysis revealed DNA methylation and transcriptome signatures, linking epigenetic silencing to phenotypic manifestation. C22.5: SIRT6 knockdown in Drosophila neurons reduced movement; in neuroblasts, it was lethal, highlighting its role in neurodevelopment. C22.6: Long-read genome sequencing improved diagnostic sensitivity, identified ZFHX3 GGC-repeat expansion as SCA4 cause, and demonstrated abnormal autophagy in affected cells.
medpagetoday.com
·

Can Immunotherapy Go the Distance in ES/LS Small-Cell Lung Cancer?

Accelerated FDA approvals bring hope for small-cell lung cancer (SCLC) treatments like tarlatamab, though caution is advised due to potential withdrawal risks. The NRG Oncology/Alliance LU005 trial found no survival benefit from adding atezolizumab to standard chemoradiotherapy in LS-SCLC, but twice-daily radiation showed improved survival.
pharmacytimes.com
·

Pelabresib and Ruxolitinib Achieve Primary End Point in Phase 3 MANIFEST-2 Trial

The MANIFEST-2 trial (NCT04603495) showed pelabresib (CPI-0610; Novartis) plus ruxolitinib (Jakafi; Incyte Corp) improved spleen volume, total symptom score (TSS), anemia, and bone marrow fibrosis (BMF) in JAK inhibitor-naïve myelofibrosis (MF) patients compared to ruxolitinib and placebo. At week 24, 65.9% achieved SVR35, and at week 48, 56.5% achieved SVR35 with pelabresib and ruxolitinib, versus 37.5% with placebo and ruxolitinib. The data will be presented at the 66th ASH Annual Meeting and Exposition on December 8, 2024.
drugs.com
·

Genes Highlight Who'll Benefit From Multiple Myeloma Treatment With Venclexta

Genetic tests identify patients with multiple myeloma likely to respond to Venclexta, with a six-gene pattern predicting response. Venclexta, approved in 2018, inhibits BCL-2 to restore natural cell death. Combining Venclexta with CDK7 inhibitors may enhance patient response, with further studies planned.
drugs.com
·

Genes Highlight Who'll Benefit From Multiple Myeloma Therapy

Genetic tests can predict which multiple myeloma patients will respond to Venclexta, with a six-gene pattern indicating likelihood of response. Combining Venclexta with CDK7 inhibitors may improve outcomes, especially for patients less responsive to Venclexta alone.
nature.com
·

AAV delivery strategy with mechanical support for safe and efficacious cardiac gene transfer in swine

Yorkshire pigs (3 months old) were used in studies involving MCS hemodynamic, AAV, and gold nanoparticle delivery after acute MI. Procedures adhered to IACUC guidelines, with pigs acclimatized for 72 hours. MCS assessed hemodynamics during coronary occlusions, while AAV6.Luc was delivered via intracoronary or intramyocardial methods, with MCS used in some groups. Gold nanoparticles were injected similarly, followed by heart explantation for analysis. Tissue harvest included LV sections and extra-cardiac tissues for expression analysis. Echocardiogram and hemodynamic data were collected, with viral genome and luciferase expression quantified. Electron microscopy analyzed gold nanoparticle distribution. Statistical analysis used Student’s t-test, Kruskal-Wallis test, and Spearman’s correlation test.
drugs.com
·

Scientists Find Way to Deliver Medicines Across Brain's Protective Barrier

Researchers developed a technique, the blood-brain barrier-crossing conjugate (BCC) system, to deliver genetic therapies into mouse brains, reducing harmful gene activity linked to ALS, Alzheimer’s, and dementia. The BCC10 compound uses transcytosis to transport drugs across the blood-brain barrier safely and efficiently.

New Platform Overcomes Blood-Brain Barrier for Drug Delivery

Researchers developed a blood-brain barrier-crossing conjugate (BCC) system using γ-secretase-mediated transcytosis to deliver large therapeutic molecules into the brain via intravenous injection, effectively silencing harmful genes in ALS and Alzheimer’s models without significant side effects, potentially revolutionizing treatments for neurological and psychiatric disorders.
news-medical.net
·

Hamilton and Amabel James Center for AI and Human Health launches at Mount Sinai

Mount Sinai Health System opens the Hamilton and Amabel James Center for AI and Human Health, focusing on enhancing healthcare delivery through AI research and development. The 12-story, 65,000-square-foot facility houses interdisciplinary teams combining AI with data science and genomics, aiming to revolutionize diagnosis and treatment. Supported by a gift from Hamilton and Amabel James, the center includes the Windreich Department of AI and Human Health, Hasso Plattner Institute for Digital Health, Institute for Genomic Health, Biomedical Engineering and Imaging Institute, and Institute for Personalized Medicine.

New Method Shows Promise to Deliver Therapeutics Across the Blood-Brain Barrier

Mount Sinai researchers developed a platform to safely deliver large therapeutic molecules across the blood-brain barrier, potentially treating ALS, Alzheimer’s, and addiction via γ-secretase-mediated transcytosis and BCC10 conjugates.
© Copyright 2024. All Rights Reserved by MedPath