Off-target effect– tag –
-
siRNA Therapy Successfully Switches Off Harmful Genes: FDA-Approved Patisiran and Other Drugs for Hereditary Amyloidosis
Works in Progress Magazine UK Overview siRNA therapy is emerging as a promising treatment to slow or halt disease progression by temporarily silencing specific genes. For conditions like hereditary transthyretin amyloidosis, FDA-approved... -
Comprehensive Review of CRISPR Gene Therapy: Potential Applications and Challenges for Sickle Cell Disease and Beta-Thalassemia
International Journal of Basic & Clinical Pharmacology Global Overview A comprehensive review covered the discovery, mechanism, therapeutic applications, and current limitations of CRISPR-Cas9, a revolutionary genome editing technology t... -
2026 ‘In Vivo CRISPR Medicine’ Symposium to Focus on Commercialization and Scalable Deployment of In Vivo Genome Editing
CRISPR Medicine News (CMN x CSGCT Virtual Symposium) USA Overview The upcoming 'In Vivo CRISPR Medicine: From Scientific Promise to Patients at Scale' virtual symposium on September 3, 2026, will highlight the transition of in vivo genom... -
Fatal Adverse Event Halts HuidaGene’s First-in-Human CRISPR Gene Therapy Trial for DMD
HuidaGene Therapeutics China Overview HuidaGene Therapeutics has reported a fatal serious adverse event (SAE) in the HG302-01 first-in-human trial of its investigational CRISPR-based gene-editing therapy, HG302, for Duchenne muscular dys... -
Researchers Uncover Key Cellular Uptake Pathway for Antisense Therapies, Paving Way for Enhanced Efficacy
ecancer Global Overview Researchers have made a significant discovery, identifying the crucial intracellular uptake pathway for antisense oligonucleotides (ASOs), a therapeutic approach to halt aberrant protein production in various dise... -
AI-Driven CRISPR Design: Revolutionizing Gene Editing with Enhanced Precision and Efficiency
Preprints.org International Overview A comprehensive review systematically investigates AI-driven tools and methodologies for CRISPR design, highlighting AI's role in significantly enhancing gene editing precision, efficiency, and automa... -
bioRxiv Unveils Joint Transcriptomic and Morphological Phenotype Modeling for Generative Molecular Design
bioRxiv International Overview A bioRxiv preprint details research on jointly modeling transcriptomic and morphological phenotypes for generative molecular design. This novel approach allows for the simultaneous consideration of gene exp... -
In Vivo CAR-T Cell Therapy Achieves Clinical Proof of Concept; AI Integration Poised to Revolutionize Cancer Treatment
OncoDaily Unknown Overview In vivo CAR-T cell therapy has achieved initial clinical proof of concept, demonstrating its potential to transform cancer treatment. This novel approach directly delivers and expresses CAR genes in T-cells wit... -
CRISPR Cancer Therapy 2026: 32 Clinical Trials Underway, Gene-Edited T-Cells Face Efficacy and Off-Target Challenges
Hirschfeld Oncology USA Overview As of 2026, 32 clinical trials are investigating CRISPR-edited immunotherapies for cancer, predominantly focusing on ex vivo gene-edited immune cells. While these therapies generally demonstrate favorable... -
RNA Therapeutics Advance with Delivery Challenges: mRNA Vaccines, siRNAs, and ASOs Drive Disease Treatment
Facebook (Ambry Genetics) USA Overview Recent publications highlight the profound impact of RNA-based therapeutic modalities, including mRNA vaccines, siRNAs, and ASOs, in treating viral infections, genetic disorders, and cancer. However...
12