gene editing

CRISPR-CAS GENOME EDITING IN PHARMACEUTICAL BIOTECHNOLOGY: ADVANCES, THERAPEUTIC APPLICATIONS, CHALLENGES AND FUTURE PERSPECTIVES

CRISPR-Cas genome editing has evolved from a bacterial adaptive immune mechanism into a validated pharmaceutical platform spanning drug discovery, direct genetic therapeutics, and molecular diagnostics. This review provides a pharmaceutically oriented synthesis of the technology’s mechanistic foundations, its evolution...

Composite Cucurbitaceae Plants as a Tool to Study Physiological and Molecular Mechanisms of Root Development

Cas9 nickase-mediated gene editing enables precise large-fragment integration with minimal byproducts

Protocol for non-viral HDR-based CRISPR/Cas9 platform for small custom editing in primary T cells

CRISPR/Cas9 enables precision gene editing via homology-directed repair (HDR) for mutation correction and disease modelling. Here we present a customizable T cell single nucleotide variant (SNV) correction platform based on non-viral HDR, previously described in Mamia et al. Precision T Cell Correction Platform for...

variant-fm-benchmark: evidence strength of splice-region variant effect predictors against saturation genome editing functional assays

variant-fm-benchmark 3.0.0: code, data and every output of the study "Evidence strength of splice-region variant effect predictors assessed against saturation genome editing data in seven cancer susceptibility genes" (N. Zhang), with one command that rebuilds every output from public sources and checks it against the a...

Introduction Pages, NBHA-CN 54(3), 2026

The papers published in Notulae Botanicae Horti Agrobotanici Cluj-Napoca, Issue 3, Volume 54, 2026, represent exciting new research in different topics of life science, specifically in plant science, horticulture, agronomy, crop science, and forestry. Among the interesting articles, we invite you to find news about: Ge...

Wild species and landraces of sorghum for biotic and abiotic stress resilience: Opportunities and future prospects

Intensive domestication and continuous selection have substantially narrowed the genetic diversity of cultivated sorghum (Sorghum bicolor (L.) Moench), limiting its adaptive capacity to emerging biotic and abiotic stresses associated with climate change. Crop wild relatives (CWRs) and traditional landraces represent va...

The C-terminus of the KREH1 helicase is important for RNA binding and association with mitochondrial RNA editing complexes in T. brucei

Mitochondrial pre-mRNA editing in kinetoplastids, a clade that includes human-infective parasitic protists such as Trypanosoma and Leishmania, is required to produce translatable, functional gene products from otherwise nonfunctional mitochondrial precursor transcripts of essential respiratory chain complex subunits. R...

Generation of lachrymatory factor synthase-suppressed onion (Allium cepa L.) by Agrobacterium-mediated gene transfer for CRISPR/Cas9 genome editing

Introduction Lachrymatory factor, an irritating volatile compound that induces tearing, is produced when onion bulbs are cut or chopped. Lachrymatory factor synthase (LFS)-specific suppression in onion may reduce the lachrymatory effect and enhance flavor and health-functional properties. Genome editing enables precise...

Mutation-specific correction of SOD1 in familial ALS using prime and base editing in human induced pluripotent stem cells

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron degeneration and respiratory failure. Approximately 10% of ALS cases are familial, with mutations in SOD1 representing a major genetic cause. Preservation of physiological SOD1 function may be i...

Managing Trait Trade-Offs in Tomato Breeding Through Genome Editing

Tomato improvement often involves trade-offs between fruit yield and quality, sweetness and fruit size, disease resistance and growth, reproductive performance and stress adaptation, and shelf life and flavour. Although CRISPR-Cas-mediated genome editing enables targeted modification of tomato genes, DNA-level precisio...

CRISPR-CAS GENOME EDITING IN PHARMACEUTICAL BIOTECHNOLOGY: ADVANCES, THERAPEUTIC APPLICATIONS, CHALLENGES AND FUTURE PERSPECTIVES

CRISPR-Cas genome editing has evolved from a bacterial adaptive immune mechanism into a validated pharmaceutical platform spanning drug discovery, direct genetic therapeutics, and molecular diagnostics. This review provides a pharmaceutically oriented synthesis of the technology’s mechanistic foundations, its evolution...