gene editing

Mitochondrial Quality Control in Inherited Mitochondrial Cardiomyopathy: Convergent Pathobiology and a Testable Therapeutic Framework

Inherited mitochondrial cardiomyopathies arise from pathogenic variants affecting oxidative phosphorylation, mitochondrial DNA maintenance, cardiolipin remodeling, protein import, cofactor metabolism, and mitochondrial dynamics or proteostasis. These disorders may be cardiac-predominant or part of multisystem disease....

Genetic principles enabling the creation of bacteria-based therapeutics.

This review examines the genetic design principles and liabilities of bacteria-based therapeutics, approaches used to produce clinical and near-clinical candidates, and the emergence of programmed bacteria delivering complete gene editing systems to therapeutically relevant cells.

Fusion of a non-specific DNA-binding domain enhances Cas12a trans-cleavage for robust nucleic-acid diagnostics

CRISPR–Cas12a underlies powerful genome-editing and nucleic-acid detection technologies, yet its performance is limited by inefficient target engagement and low catalytic turnover, particularly at low target abundance and elevated temperatures. Here, we report a modular protein-engineering strategy to enhance Cas12a tr...

Improved long-term proliferation and myogenic differentiation of bovine muscle stem cells through CDKN2A gene editing

ChoRS: a 158-gene choline reprogramming score for MASLD - analysis code and derived tables (v1.0.0)

Analysis code and derived result tables for a multi-cohort, in-silico study quantifying choline-glycerophospholipid reprogramming across the metabolic dysfunction-associated steatotic liver disease (MASLD) spectrum. Score definition. ChoRS is a frozen set of 158 unique genes in five non-overlapping modules: M1 Kennedy...

ChoRS: a 158-gene choline reprogramming score for MASLD - analysis code and derived tables (v1.0.0)

Analysis code and derived result tables for a multi-cohort, in-silico study quantifying choline-glycerophospholipid reprogramming across the metabolic dysfunction-associated steatotic liver disease (MASLD) spectrum. Score definition. ChoRS is a frozen set of 158 unique genes in five non-overlapping modules: M1 Kennedy...

Abscisic Acid-Mediated Drought Tolerance in Populus: Physiological, Molecular, and Biotechnological Perspectives

Current knowledge of ABA signaling pathways and their roles in drought resilience in Populus is summarized, covering physiological responses, molecular regulatory networks, hormonal crosstalk, biotechnological advances, and emerging field applications.

ADAR1 and ADAR2 Expression in the Thoracic Aortic Wall Correlates with Aneurysm Severity and Dissection Risk: Insights into A-to-I RNA Editing Dysregulation in Marfan Syndrome-Derived vSMCs

Findings establish ADAR enzymes as candidates tissue biomarkers of TAA severity and ADAR1 as the principal driver of epitranscriptomic dysregulation in MFS aortopathy.

Structure-guided discovery and engineering of miniature CRISPR-Cas12m for epigenome editing.

Protocol for robust gene knockout and reliable validation in human cell lines using quad-guide RNA vectors

This strategy provides an efficient workflow for gene knockout that is rapidly confirmed through PCR amplification of mRNA derived from the targeted gene loci that is rapidly confirmed through PCR amplification of mRNA derived from the targeted gene loci.

Using Artificial Intelligence (AI) and Bioprocessing for Drop-in Fuel

Across global energy systems, the adoption of drop-in fuels offers a practical pathway to decarbonization while preserving the value of existing petroleum infrastructure. Their molecular similarity to fossil fuels eliminates the compatibility issues that hindered earlier biofuels such as ethanol and biodiesel. As a res...

Orthotopic liver xenotransplantation from gene-modified pig to decedent human.