Skip to content

Category

gene editing

268 papers

#gene editing Review Open access Aug 2026

Immune cell development from human pluripotent stem cells.

The landmark derivation of induced pluripotent stem cells (iPSCs) sparked an enormous range of research on development of diverse cell populations. Perhaps no aspect of this work has been as productive as studies on blood and immune cell production. Indeed, essentially all human blood cell populations can be derived from human iPSCs. While derivation of transplantable hematopoietic stem cells (HSCs) from iPSCs has been relatively challenging, clinical translation of iPSC-derived immune cells has been particularly productive. Notably, more patients have been treated with iPSC-derived natural killer (NK) cells than any other iPSC-derived cell type. Use of iPSCs provides a key platform to incorporate multiplexed gene edits before differentiation, enabling NK cells to be engineered with tumor-targeting CARs, cytokine support for improved persistence, enhanced tumor trafficking, and resistance to host immune rejection, modifications uniformly expressed across the entire cellular product. While T cells, macrophages and other immune cells can be produced from human iPSCs, iPSC-derived NK cells have been the most widely used in clinical trials for treatment of refractory malignancies, as well as autoimmune disease. This review summarizes research with a focus on clinical translation of iPSC-derived immune cells, as well as highlights continued challenges and prospects of this field.

Luisjesus S Cruz, Alejandro R. Castañeda, Dan S Kaufman · 0 citations
#gene editing Review Open access Aug 2026

Mitochondrial DNA A3243G variant-associated MELAS: Recent advances in clinical trials, therapeutic interventions, and disease-modifying strategies.

The therapeutic landscape for MELAS is evolving from symptomatic care toward mechanism-based disease modification, led by taurine as the first regulatory-approved disease-modifying therapy and complemented by late-stage small molecules and mitochondrial genome-editing technologies.

Kuan-Yu Chu · 0 citations
#gene editing Review Open access Aug 2026

Beyond Mixed Utilization: Fractionation, Functional Divergence, and Precision Applications of Soybean 7S and 11S Globulins.

Soybean β-conglycinin (7S) and glycinin (11S) differ markedly in their structure, physicochemical properties, and bioactivity; however, industrial practice still treats them as a mixed ingredient, obscuring their differentiated functional potential. This review critically evaluates fractionation technologies through the lens of the "purity-yield-sustainability" trilemma. First-generation chemical precipitation methods achieve high purity at the laboratory scale but suffer from heavy reagent use, environmental burden, and poor scalability. Second-generation green and physical techniques, such as phytase-assisted, membrane-based, and field-assisted separation, improve sustainability but face challenges in fouling control, process stability, and scale-up. Third-generation upstream strategies, including breeding and gene editing, fundamentally alter the 7S/11S ratio at the source, potentially bypassing downstream tradeoffs. Functionally, 7S globulin excels in regulating lipid metabolism and reducing obesity and non-alcoholic fatty liver disease, whereas 11S globulin shows advantages in blood pressure regulation and cardiovascular protection. Structurally, 7S exhibits favorable emulsification and foaming capacities, whereas 11S dominates gel network formation, supporting diverse applications from plant-based foods to nanocarriers and biodegradable films. Future breakthroughs lie in AI-guided hybrid separation systems, data-driven process optimization, and direct linking of fractionation outcomes to end-use functionality. Moving beyond mixed utilization toward precision deployment, soybean 7S and 11S globulins can evolve from bulk commodities into high-value resources for sustainable food systems and precision health applications.

Shuyin Hu, Yuyang Huang, Ya-Qi Lu et al. · 0 citations
#protein folding Open access Aug 2026

Multi-species Comparative Analysis Identifies Conserved Enhancers for Improving Casein Expression via Gene Editing.

This research introduces a novel strategy to augment casein expression in ruminants, providing a significant theoretical foundation and technical support for the precision breeding of high-casein dairy cows and goats.

Zhenliang Zhu, Xiaoyu Mi, Wei Yu et al. · 0 citations
#gene editing Review Open access Aug 2026

ZBP1 in Neuroinflammation and Neurodegeneration: Z-Nucleic-Acid Sensing, RHIM Signalling and Therapeutic Targeting.

Competition with adenosine deaminase acting on RNA 1 (ADAR1), melanoma differentiation-associated protein 5 (MDA5), double-stranded RNA-dependent protein kinase (PKR), the cyclic guanosine monophosphate–adenosine monophosphate–adenosine monophosphate synthase–stimulator of interferon genes (cGAS–STING) pathway, and other nucleic-acid-sensing

Matei Șerban, C. Toader, Razvan-Adrian Covache-Busuioc · 0 citations
#gene editing Review Open access Aug 2026

Application of Lipid Nanoparticles (LNPs) in Nucleic Acid Delivery and Precision Medicine

This article systematically reviews the component design, structural engineering, preparation technologies, clinical applications, and future development directions of LNPs, aiming to provide theoretical support and practical guidance for vector development in the era of precision medicine.

Jin-Yuan Hu · 0 citations
#gene editing Open access Aug 2026

Genetic transformation and CRISPR/Cas12a-mediated gene editing of European beech (Fagus sylvatica L.) employing a transient protoplast system

A new protoplast-based platform enables transient transformation and proof-of-concept CRISPR/Cas12a-mediated genome editing in European beech, a key but recalcitrant forest tree species.

Virginia Zahn, Alice-Jeannine Sievers, B. Kersten et al. · 0 citations
#gene editing Review Open access Aug 2026

Recent applications of CRISPR-Cas systems in animal models of human cardiovascular disease: Review article

A review of studies in which the creation of a CRISPR-Cas9 animal model has been used to find genetic drivers and develop therapies across 4 CVD domains shows the vast range of gene editing applications and the value of models that parallel human genetic pathophysiology for specific disease processes.

J. Longmire, Harrison Smith-Jaoudi, Eesha Balar et al. · 0 citations
#gene editing Open access Aug 2026

Viperin as a putative antiviral effector in rainbow trout (Oncorhynchus mykiss) cells identified by CRISPR-Cas9 functional analysis following exposure to inactivated viral hemorrhagic septicemia virus

It is demonstrated that viperin plays a central role in coordinating antiviral responses in rainbow trout macrophages and is essential for proper activation of classical IFN–ISG pathways.

Mariana Vaz, A. Florea, G. E. Themudo et al. · 0 citations
#gene editing Open access Aug 2026

The gene homologous to OsCd1, designated as ABNORMAL SHOOT IN YOUTH (OsASY), functions as a cadmium influx transporter in rice.

Preliminary evidence indicates that targeted editing of OsASY offers a promising strategy to reduce grain Cd content without adversely affecting major agronomic traits or yield, highlighting its potential as a molecular target for breeding low-Cd-accumulating rice varieties.

C. Tu, Wei Huang, Daobo Wang et al. · 0 citations

From tech blogs

See all →
MIT News · Artificial Intelligence Aug 17, 2026

Q&A: Rethinking how innovation happens

In his latest book, Professor Eugene Fitzgerald examines the forces that turn breakthroughs into value — and why innovation resists simple formulas.