gene editing

The emerging role of ARE1 in the molecular regulation of nitrogen use efficiency: opportunities for genome editing

Inhibition of MHC class II CLIP binding by HLA gene editing

The Role of SOD1 and Oxidative Stress in Amyotrophic Lateral Sclerosis: An Overview

Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disorder that selectively affects motor neurons. The median survival time after diagnosis is 2 to 5 years. Oxidative stress (OS), driven mainly by reactive oxygen species (ROS), plays a central role in motor neuron (MN) degeneration. In this...

Collaborating with AIProSaB-A researcher's perspective on patient advocacy for progeria.

Progeria is an extremely rare genetic disease in which affected individuals age rapidly due to a specific mutation in the LMNA gene, which results in the production of progerin, an abnormal protein that damages cell nuclei and causes progressive multi-organ disease. AIProSaB is an Italian association founded by Sammy B...

The RNA helicase DDX1 modulates intraerythrocytic development and gene expression in Plasmodium falciparum

RNA helicases participate in multiple aspects of RNA metabolism, but the functions of most helicases in malaria parasites remain poorly understood. PfDDX1 is a conserved DEAD-box RNA helicase whose role during the asexual blood-stage development of Plasmodium falciparum has not been defined. Endogeno...

Gene Therapy: Routes of Inner Ear Delivery

Abstract: Gene delivery and the various routes of administration used to deliver viral vectors constitute a rapidly evolving field, with recent success in restoring hearing in children with OTOF variants in ongoing clinical otoferlin trials. With the rise of gene therapy for major hereditary hearing disorders, both syn...

Hybridization-Mediated Reversible sgRNA Caging Enables Tumor-Selective Gene Editing for Potentiating Antitumor Immunity via cGAS Demethylation.

Spatiotemporal precision remains a critical challenge against the gene editing technology used for disease treatment. We herein developed a tumor cell-selective CRISPR/Cas9 gene editing system by introducing a complementary single-stranded DNA (ssDNA) containing apurinic/apyrimidinic (AP) sites (AP-ssDNA) along the tar...

Uncovering early-stage vaccine candidates against Fasciola hepatica through integrative comparative proteomic analysis and proof-of-concept CRISPR-Cas9 gene editing

Fasciolosis, mainly caused by the liver fluke Fasciola hepatica , is a globally distributed parasitic disease that affects both livestock and humans, generating significant economic losses and public health concerns. Current control strategies rely mainly on anthelmintic treatments, but the increasing emergence of drug...

Dr. Dharmesh R. Patel 1, Dr. Khushbu S. Rana 2 and Dr. I. H. Kalyani 3

Rabies remains one of the most lethal zoonotic neurotropic diseases affecting warm-blooded animals and humans, causing an estimated 59,000 human deaths annually despite being completely preventable. Caused by neurotropic single-stranded negative-sense RNA viruses of the genus Lyssavirus, family Rhabdoviruses, the Rabie...

A base-editing and auxotrophic selection platform for linking epigenetic regulators to silent biosynthetic gene clusters in Aspergillus nidulans

Filamentous fungi harbor numerous silent biosynthetic gene clusters (BGCs), representing an underexplored reservoir of natural products. Chemical inhibition of epigenetic regulators is an effective strategy for activating silent BGCs, but it often lacks specificity and cannot readily link activated clusters to their co...

A CRISPR-Cas13 system based diagnostic tool for arboviruses

The unpredictability of the emergence and re-emergence of arboviruses underscores the need for enhancing global preparedness in surveillance capacity for tracking and predicting the growth of arboviral epidemics and case-specific arboviral disease management. In particular, the development of a more advanced clinical d...

Precision Therapeutics for Monogenic Epilepsies: From Molecular Mechanisms to Disease Modification in Developmental and Epileptic Encephalopathies and Neurodevelopmental Disorders with Epilepsy

Precision therapeutics are shifting the treatment paradigm for monogenic DEEs and NDD-E from empiric seizure control toward mechanism-based targeting of dysregulated molecular pathways and gene-based disease-modifying therapies.