Improved gene targeting and nucleic acid carrier molecule, in particular for use in plants

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes a novel nucleic acid carrier molecule—essentially a customizable fusion protein—that can simultaneously specifically bind a donor nucleic acid (the genetic material to be inserted) and a target nucleic acid sequence (the genomic insertion site in an organelle, such as the nucleus, chloroplast, or mitochondrion). By bringing the donor DNA in close proximity to the target site, it facilitates efficient, targeted gene editing or insertion, particularly in plants, but the approach is adaptable for other eukaryotic or prokaryotic systems. The invention further outlines methods and compositions for using these molecules to improve gene targeting, transformation, and precise genetic modification in living cells.

Use CasesContent extracted from patent full text and abstract with AI.

• Precise genetic modification and trait improvement in crop plants by inserting or modifying genes at targeted locations.
• Correction of genetic defects in plant cells for research or agricultural purposes.
• Targeted genome editing in animal or human cells for therapeutic gene correction or disease modeling (e.g., correcting mutations or introducing beneficial alleles).
• Efficient transgene insertion into organelle genomes (such as mitochondria or chloroplasts), which are typically hard to edit.
• Delivery of specific RNAs (like miRNA, RNAi, or antisense RNA) to chosen genomic sites for gene silencing or functional studies.
• Disabling integrated viral sequences (like HIV) in host genomes by targeted editing or insertion/deletion of specific DNA.
• Development of gene therapy applications in animal and human medicine where targeted correction or insertion is needed.
• Creation of genetically modified organisms (plants, animals, microbes) with minimal off-target genetic changes.
• Synthetic biology applications, such as building biological circuits by precisely assembling DNA in living systems.

BenefitsContent extracted from patent full text and abstract with AI.

• Dramatically increases the frequency and precision of gene targeting, especially in plants where it is historically inefficient.
• Reduces unwanted or random integration of genetic material, thereby improving safety and predictability of genetic engineering.
• Enables editing of challenging genomic locations, such as organelle DNA (chloroplasts/mitochondria), which are typically resistant to traditional transformation methods.
• Allows flexible design by using various sequence-specific DNA/RNA binding domains (e.g., TAL effectors, zinc fingers, Cas9, custom transcription factors), making it adaptable for different targets and species.
• Facilitates proximity between donor and target nucleic acids, greatly enhancing homologous recombination or targeted insertion efficiency.
• Improves transformation efficiency and reduces technical requirements for generating edited organisms.
• Enables precise gene therapy approaches, including repair of disease-related genes or deactivation of integrated viruses, with lower risk of off-target effects.
• Supports delivery of a wide range of nucleic acids (DNA, RNA, labeled nucleotides) for diverse applications including editing, marking, and therapeutic intervention.
• Potential for use in synthetic biology, enabling assembly or rewiring of genetic circuits in living cells with high accuracy.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

The present invention relates to a nucleic acid carrier molecule, comprising the general formula M-S 1 -L-W-S 2 , wherein M is a first polypeptide specifically binding to a donor nucleic acid sequence to be transferred into an organelle of a cell, W is a second polypeptide specifically binding to a target nucleic acid sequence, wherein said target nucleic acid sequence is located in an organelle of a cell, L is missing or is linking group allowing M and W flexibility and semi-independence, and S 1 and S 2 independently of each other are missing or are a signal peptide sequence, and can be fused to M and W proteins either N-or C-terminally, wherein said donor nucleic acid sequence is brought into close proximity with said target nucleic acid sequence when both nucleic acid sequences are bound to said carrier molecule. The present invention furthermore relates to methods for recombinantly transforming a nucleic acid into an organelle in a cell, preferably a plant cell, employing said nucleic acid carrier molecule.