C12N9/226

USE OF ISCB IN GENOME EDITING
20250283116 · 2025-09-11 ·

Provided are modified proteins that are functional in RNA-guided DNA cleavage. The proteins include modified IscBs protein that have a modification of the N-terminus or C-terminus, or both. The modifications include a truncation of a PLMP domain of the IscB protein, or a PLMP domain that is relocated to a position of the IscB protein that is not the N-terminus. The modified IscB protein can be provided as a component of a fusion protein. The modified IscB proteins are used with an RNA to modify a DNA substrate.

TRANSGENIC ANIMAL HAVING MODIFIED MYOSTATIN GENE

The present application relates to an animal or cell having a myostatin gene in which 12 base pairs of the second exon are deleted. The present application may also comprise a composition capable of manipulating the deletion of 12 base pairs of a myostatin gene to construct the animal or the cell. The present application also relates to use of the composition for increasing muscle.

METHODS AND SYSTEMS FOR DETECTING A TARGET USING BRCAS12B AND GENETICALLY ENGINEERED VARIANTS THEREOF
20250283182 · 2025-09-11 ·

Novel genetically engineered thermostable Cas12b enzymes with greater thermal stability, higher melting point and/or increased trans-cleavage activity than wild type Cas 12b enzymes are provided. Methods, systems, and kits for one-pot detection of target polynucleotide are also provided that combine isothermal amplification with CRISPR-based detection with thermostable Cas enzymes in a single reaction vessel.

COMPOSITIONS AND METHODS FOR INCREASING DELETION EFFICIENCY OF NUCLEIC ACID SEGMENT BY MODULATION OF NHEJ REPAIR PATHWAY
20250283113 · 2025-09-11 ·

The present invention relates to a composition and method for increasing deletion efficiency of nucleic acid segments in a target gene by modulation of non-homologous end joining (NHEJ) repair pathway. Specifically, the present invention relates to a CRISPR/Cas system (for example, CRISPR/Cas12f1 system) for increasing deletion efficiency of nucleic acid segments in a target gene by modulation of non-homologous end joining repair pathway.

USE OF TRANSDIFFERENTIATION OF GLIAL CELLS INTO NEURONS IN PREVENTION OR TREATMENT OF DISEASES ASSOCIATED WITH NEURON LOSS-OF-FUNCTION OR DEATH
20250281648 · 2025-09-11 ·

The transdifferentiation of glial cells into neurons is useful for prevention or treatment of a disease associated with loss of function or death. The present disclosure relates to the field of biomedicines. More specifically, the present disclosure relates to use of a REST inhibitor in the treatment of a disease associated with loss of function or death of neurons. The present disclosure can effectively induce transdifferentiation of astrocytes into dopamine neurons by inhibiting the expression, content or activity of a gene of REST or an RNA thereof or an encoding protein thereof in astrocytes in the brain, and can effectively induce transdifferentiation of Mller glia (MG) into retinal ganglion cells (RGCs) or photoreceptor cells by inhibiting the expression, content or activity of the gene of REST or the RNA thereof or the encoding protein thereof in the retina, thereby preventing and/or treating the disease associated with loss of function or death of neurons.

ENHANCED CELL-FREE BACTERIOPHAGE SYNTHESIS BY GENETIC MODULATION OF BACTERIAL TRANSCRIPTION/TRANSLATION MACHINERY (TXTL) MACHINERY

The present disclosure relates to compositions including or obtained from genetically modified bacterial host cells (e.g., E. coli) and methods for using the same for cell-free bacteriophage synthesis (CFBS). In particular, the present technology relates to genetically modified E. coli that overexpress one or more of translation initiation factor IF-3 (infC), OxyS and CyaR and/or repress RecC subunit exonuclease RecBCD, and methods for using the same to obtain improved CFBS yields.

COMPOSITIONS, METHODS AND USES FOR TREATING CYSTIC FIBROSIS AND RELATED DISORDERS

Described herein are compositions, kits, and methods for potent delivery to a cell of a subject. The cell can be of a particular cell type, such as a basal cell, a ciliated cell, or a secretory cell. In some cases, the cell can be a lung cell of a particular cell type. Also described herein are pharmaceutical compositions comprising a therapeutic or prophylactic agent assembled with a lipid composition. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Further described herein are high-potency dosage forms of a therapeutic or prophylactic agent formulated with a lipid composition.

Compositions and Methods for RNA Editing
20250283112 · 2025-09-11 ·

The present disclosure provides a hybrid RNA molecule comprising a targeting region and a donor RNA, and compositions comprising the hybrid RNA molecule. A hybrid RNA molecule of the present disclosure is useful in methods of modifying a target RNA, which methods are also provided.

ENGINEERING B CELL-BASED PROTEIN FACTORIES TO TREAT SERIOUS DISEASES
20250283036 · 2025-09-11 ·

The invention(s) disclosed herein relate to improved methods for expanding cell populations, particularly B cell populations. The invention further relates comprising improved cell media, compositions thereof, and methods of using such expanded B cells. Wherein a population of cells comprises engineered human B cells, wherein the engineered human B cells comprise a therapeutic protein, whose gene has been inserted into the beta-2M locus.

CONFERRING CYTOPLASMIC MALE STERILITY

Provided herein are methods for conferring cytoplasmic male sterility (CMS) on a plant line. The methods include obtaining a first plant comprising a CMS cytoplasm that is also a haploid inducer (CHIP) and crossing it with a second plant that comprises a desired nuclear genome (DIP). The CHIP also comprises a cenh3 mutation and may contain an anthocyanin marker and a restorer factor. The method further comprises generating progeny from said cross. The progeny produced from the cross of the method is haploid and comprises the CMS cytoplasm of the CHIP as well as the desired nuclear genome of the DIP. The progeny further lacks any anthocyanin marker or restorer factor.