Patent classifications
C12N2750/00043
ANELLOVECTORS AND METHODS OF USE
This invention relates generally to anellovectors and compositions and uses thereof.
COMPOSITIONS AND METHODS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY
It has been discovered that overexpression of DLC1-i1 partly rescues neuronal formation and apoptosis of neuromuscular cells in subjects with spinal muscular atrophy (SMA). Since loss of DLC1-i1 confers specific MN defects in SMA, compositions and methods thereof for the treatment of SMA by delivery of AAV-SYN-DLC1-i1 into the neuronal population of subject with SMA are provided. The methods improve survival and restore locomotion ability to a greater extent than that of commercially-available compositions and methods. Methods including administration of both DLC1-i1 and SMN1 transgenes provide synergistic effects in improving the survival and enhancing locomotion ability in subject with SMA.
ANELLOVIRUS CONSTRUCTS COMPRISING RECOMBINATION SITES AND CROSS-PACKAGING OF ANELLOVECTOR PARTICLES AND GENETIC ELEMENTS
This invention relates generally to modified anellovirus capsid proteins, anellovectors, anelloVLPs, and compositions and uses thereof.
MYCOVIRUS-INDUCED GENE SILENCING VECTOR, CONSTRUCTION METHOD AND APPLICATION THEREOF
A mycovirus-induced gene silencing vector and a construction method and an application thereof are provided. A nucleotide sequence of the mycovirus-induced gene silencing vector is shown in SEQ ID NO: 2, and construction method for the mycovirus-induced gene silencing vector includes: (1) connecting three single-stranded circular DNA molecules DNA-A, DNA-B and DNA-C of the mycovirus FgGMTV1/HB58 in series and introducing them into a same vector to construct a recombinant vector; and (2) carrying out a deletion mutation on a coding protein p26 of the DNA-C molecule in the recombinant vector to obtain the mycovirus-induced gene silencing vector.
Mycovirus-induced gene silencing vector, construction method and application thereof
A mycovirus-induced gene silencing vector and a construction method and an application thereof are provided. A nucleotide sequence of the mycovirus-induced gene silencing vector is shown in SEQ ID NO: 2, and construction method for the mycovirus-induced gene silencing vector includes: (1) connecting three single-stranded circular DNA molecules DNA-A, DNA-B and DNA-C of the mycovirus FgGMTV1/HB58 in series and introducing them into a same vector to construct a recombinant vector; and (2) carrying out a deletion mutation on a coding protein p26 of the DNA-C molecule in the recombinant vector to obtain the mycovirus-induced gene silencing vector.
Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
The present teachings disclose nucleic acid cassettes for expressing in an insect cell a plurality of polypeptides encoded by a gene comprising overlapping open reading frames (ORFs). A cassette comprises, in 5 to 3 order, a) a first insect cell-operable promoter, b) a 5 portion of a gene comprising a first ORF of the gene, c) an intron comprising a second insect cell-operable promoter, and d) a 3 portion of the gene comprising at least one additional ORF. Vectors and insect cells comprising the cassettes are also disclosed, as well as methods for production of recombinant adeno-associated virus in insect cells using the cassettes.
Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
The present teachings disclose nucleic acid cassettes for expressing in an insect cell a plurality of polypeptides encoded by a gene comprising overlapping open reading frames (ORFs). A cassette comprises, in 5 to 3 order, a) a first insect cell-operable promoter, b) a 5 portion of a gene comprising a first ORF of the gene, c) an intron comprising a second insect cell-operable promoter, and d) a 3 portion of the gene comprising at least one additional ORF. Vectors and insect cells comprising the cassettes are also disclosed, as well as methods for production of recombinant adeno-associated virus in insect cells using the cassettes.
ANELLOVECTORS FOR DELIVERY OF EFFECTORS TO THE CENTRAL NERVOUS SYSTEM
This invention relates generally to Anelloviridae family vectors (e.g., anellovectors) and compositions and uses thereof.
NOVEL ANELLOVECTOR COMPOSITIONS AND METHODS
This invention relates generally to Anelloviridae family vectors (e.g., anellovectors) and compositions and uses thereof.
Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
The present disclosure relates to one or more agents, therapies, treatments, and methods of use of the agents and/or therapies and/or treatments for producing an antibody like protein (ALP) and one or more sequences of miRNA that are complementary to the mRNA of a target, viral-specific protein or proteins. Embodiments of the present disclosure can be used as a therapy or a treatment for a subject that has a condition whereby the production of the APL and decreased production of a target, viral-specific protein or proteins may be of therapeutic benefit.