12 results about "Repeat sequence" patented technology

Aspects of the disclosure relate to compositions and methods for the diagnosis and / or treatment of certain neurodegenerative diseases, for example those diseases associated with repeat-associated non-ATG (RAN) translation proteins, such as amyotrophic lateral sclerosis (ALS). In some embodiments, the disclosure relates to recombinant adeno-associated viruses (rAAVs) expressing antibodies and antigen-binding fragments thereof that bind to poly(GA) RAN proteins. In some embodiments, the disclosure relates to methods of treating a RAN protein-associated disease by administering to a subject in need thereof the rAAVs.

Split-pegRNA recorder reagents and methods are provided that provide specific molecular interactions within cells induce precise genome editing that inserts an event-specific barcode sequence into the predetermined genomic locus of DNA Tape. The split pegRNAs include (a) a crRNA component, including in 5′ to 3′ order (i) a spacer sequence for a genomic locus of interest; (ii) a crRNA repeat sequence necessary for binding to a CRISPR-Cas protein; and (iii) a first RNA extension; and (b) a petracrRNA component, including in 5′ to 3′ order (i) a second RNA extension; (ii) a petracrRNA antirepeat sequence necessary for binding to a CRISPR-Cas protein; (iii) a gRNA scaffold; wherein the crRNA component and the petracrRNA component are not covalently bound to each other.

A method for improving the replication of covalent closed circular plasmids is provided. [Solution] The method comprises the step of preparing a covalent closed circular plasmid having a Pol I-dependent origin of replication and an insert containing a structured DNA sequence selected from the group consisting of a reverse repeat sequence, a directional repeat sequence, a homopolymer repeat sequence, a eukaryotic origin of replication, or a eukaryotic promoter enhancer sequence, wherein the structured DNA sequence is located at a distance of less than 1000 bp from the Pol I-dependent origin of replication in the direction of replication. The method also comprises the step of modifying the covalent closed circular recombinant molecule so that the Pol I-dependent origin of replication is replaced with a Pol III-dependent origin of replication, thereby improving the replication of the covalent closed circular plasmid with the resulting Pol III-dependent origin of replication. A covalent closed circular recombinant DNA molecule without antibiotic markers is also provided.

RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-2325 and compositions, methods, and uses thereof.

This invention relates to the field of biomedical technology, specifically to a rapid and sensitive detection method for the CGG repeat sequence of the FMR1 gene and its application. It includes a primer combination for detecting the CGG repeat sequence of the FMR1 gene, comprising an upstream primer with the nucleotide sequence shown in SEQ ID NO: 1 and a downstream primer with the nucleotide sequence shown in SEQ ID NO: 2. This invention optimizes the PCR detection procedure, making it more sensitive for samples with high CGG repeat counts, while also enabling accurate quantification and faster detection, significantly shortening the detection time, effectively reducing the workload of operators, and lowering detection costs.

A CRISPR-Cas system and applications thereof are provided, also related to a Cas protein, a fusion protein and a guide polynucleotide. The Cas protein has at least 50% sequence identity compared to the sequence set forth in any one of SEQ ID NOs: 1-184, 426-428. The fusion protein comprises the Cas protein fused to a protein domain and / or a polypeptide tag. The guide polynucleotide comprises a direct repeat sequence having at least 70% sequence identity to any one of SEQ ID NOs: 185-199, 245-247, 287-303, 369 and 370-373, and a guide sequence engineered to hybridize to a target nucleic acid. The CRISPR-Cas system comprises a Cas protein having at least 90% sequence identity compared to the sequence set forth in any one of SEQ ID NOs: 1-184, 426-428, or a nucleic acid encoding thereof, and a guide polynucleotide or a nucleic acid encoding thereof.

The application discloses a double-end sequencing sample mark weight method, comprising the following steps: aligning the double-end read length corresponding to the DNA fragment in the double-end sequencing sample to the position corresponding to the whole genome reference sequence respectively, the double-end read length of the fragment being at adjacent positions, obtaining the alignment information of the double-end read length, adding an auxiliary field in the alignment information of each read length, and the auxiliary field containing the starting position of the other end read length corresponding to the read length; sorting the read length according to the reference sequence site after the alignment is completed, and saving the ordered read length in a SAM file; sequentially reading the read length from the ordered SAM file, taking the read length at the same alignment position as a group of candidate read length repeats, screening the fragment repeat sequence from the candidate read length repeat sequence according to the auxiliary field; deleting the repeat sequence or marking the repeat sequence, and writing into a new SAM file, so that the application reduces the data reading throughput in the double-end sequencing sample mark weight process, reduces the running time, and improves the running efficiency.

The invention provides a method for synthesizing a DNA sequence comprising repeat units, including designing and synthesizing an extension primer and a blocking primer based on the repeat unit, performing a PCR amplification reaction by using the repeat unit (as an amplification template), the extension primer, and the blocking primer in a PCR reaction system, to obtain the DNA sequence comprising repeat units. The invention also provides a kit for this method. The method of the invention has the characteristics such as controllable copy number for repeat synthesis, simple synthesis steps, and low cost, and is very suitable for high-throughput production in industry.

The present application relates to a kind of primer group, kit and method for detecting ATXN8OS and ATXN8 gene (CTA·TAG) n And (CTG·CAG) n Trinucleotide repeat number, belong to gene detection technical field.The primer group includes first primer pair and second primer pair;The forward primer sequence of the first primer pair is as shown in SEQ ID NO.1, and the reverse primer sequence is as shown in SEQ ID NO.2;The forward primer sequence of the second primer pair is as shown in SEQ ID NO.1, and the reverse primer sequence is as shown in SEQ ID NO.3.The present application is realized by the design of two primer pairs, complementary verification is realized, detection result is comprehensive and reliable, make up the technical deficiency of one generation sequencing and two generation sequencing in long fragment, high GC content repeat sequence detection, and detection cost is low, detection cycle is short.

An encoding method and apparatus, relating to the technical field of communications, and capable of constructing Polar codes on the basis of different repeat sequence lengths to improve decoding performance. The method comprises: mapping information bits corresponding to A first bits among K first bits to A second bits among K second bits of a first sequence having a length of (N2-N1), to obtain a second sequence and perform polar encoding, to obtain a first encoded bit sequence having a length of (N2-N1), the K second bits comprising a first set of information bits and a second set of information bits; the first set of information bits comprising T most reliable bits among bits in a second reliability sequence other than a first set of pre-frozen bits, the first set of pre-frozen bits being determined on the basis of M2 and N2; and the second set of information bits comprising (K-T) most reliable bits among bits in the second reliability sequence other than the first set of information bits and a second set of pre-frozen bits, T being determined on the basis of K, M2, and N2.

The present invention relates to a polypeptide comprising a piggyBac transposase or a fragment or a derivative thereof having transposase function comprising at least one amino acid substitution. Further, the present invention relates to a transposable element comprising a piggyBac or piggyBac-like left repeat sequence and left internal repeat sequence, wherein the left internal repeat sequence comprises at least one nucleotide modification. Furthermore, the present invention relates to a kit comprising the above transposase and / or transposable element. In addition, the present invention relates to a targeting system comprising the above transposase and / or transposable element.