gene editing

Integration of MOET and CRISPR/Cas9 RNP electroporation for MSTN genome editing in caprine zygotes

Myostatin (MSTN) is a key negative regulator of skeletal muscle growth and an important candidate gene for genetic improvement of economically relevant livestock traits. CRISPR/Cas9-mediated genome editing using ribonucleoprotein (RNP) delivery offers a precise approach for targeted MSTN modification in caprine embryos...

CRISPR/Cas9-Mediated MSTN Knockout Alters Muscle Proteome and MSTN–ACVR2B Interaction in Buffalo Myoblasts

Myostatin (MSTN) is a potent negative regulator of skeletal muscle growth and differentiation. Buffalo myoblasts were isolated from the longissimus dorsi muscle and characterized by binucleate morphology and Pax7/myosin immunostaining. Myoblasts were nucleofected with MSTN guide RNA and Cas9, and three independently de...

Engineering IL‐4 resistant proinflammatory human myeloid cells for cancer immunotherapy

Adoptive transfer of proinflammatory myeloid cells enhances antitumor T‐cell responses and limits tumor growth in murine models. Previous work identified transcription factors NF‐κB p50 and STAT6 as key regulators whose inactivation enhances the inflammatory potential of murine myeloid cells. Herein, we translate murin...

Evolution of Stem Cell Therapy: From Embroyic and Adult Stem Cells to IPSCS in Disease Modelling, Cell Replacement and Precision Therapeutics

ABSTRACT Over recent years, stem cell therapy has transitioned from a potentially viable approach to an actual method of tissue repair and regeneration. This review highlights advances in the study of stem cell biology, their classification, and application in medical practice. In particular, the use of different sourc...

Evolution of Stem Cell Therapy: From Embroyic and Adult Stem Cells to IPSCS in Disease Modelling, Cell Replacement and Precision Therapeutics

ABSTRACT Over recent years, stem cell therapy has transitioned from a potentially viable approach to an actual method of tissue repair and regeneration. This review highlights advances in the study of stem cell biology, their classification, and application in medical practice. In particular, the use of different sourc...

Mapping immunocompetent tissues for safer vaccines and gene therapy: A miRNA-based strategy for mRNA-LNP platforms

Emerging Therapies in Diabetes: New Drugs and Innovative Remedies

Diabetes mellitus is a chronic metabolic disorder with a rapidly increasing global prevalence, posing significant health and economic challenges. More than 500 million adults are currently affected worldwide, with projections indicating further increases, particularly in low- and middle-income countries. The disease ma...

Genotoxicity Evaluation of Pharmaceuticals

This chapter provides a comprehensive overview of genomic safety considerations for both small molecules and cell and gene therapies. For small chemical entities, testing is essential to identify a possible genotoxic potential early in drug development. Mutations in somatic and germline cells can lead to cancer or heri...

Post-transcriptional dysregulation precedes morphological transformation in chemically induced liver cancer

DNA lesions impede RNA polymerase II, yet the transcriptional consequences of that stress are less understood than the lesions themselves. We reanalysed total RNA and matched whole genomes from 438 liver lesions across four divergent mouse backgrounds, one of them the separate species M. caroli, 428 of them induced by...

Transposon end recognition and pairing by I-F3 CRISPR-associated transposase

To develop gene therapy tools based on CRISPR-associated transposons (CASTs), it is essential to define how transposon ends are recognized and paired during transposition. Transposon 7 (Tn7)–like transposons typically contain asymmetric left- and right-end sequences that flank and define DNA cargo. However, how the tra...

CRISPR/CAS9-based gene editing in cancer therapy

Single-allele nanoscale mapping of regulatory variants.

Millions of genetic variants are linked to human disease but identifying underlying mechanisms is challenging because most variants are noncausal and lie within the noncoding genome. We developed a Micro Capture-C variant-to-function platform (MCCv) based on analysis of single-allele chromatin structure. This can ident...