摘要:
The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A.
摘要:
One aspect of the invention provides a method of contraception by administering to a female subject a composition comprising Mullerian inhibiting substance (MIS). The MIS can be produced endogenously in the subject by a vector, where the vector comprises a polynucletide encoding a recombinant MIS protein. In some embodiments, the contraception is permanent and only requires administration of the composition once. Another aspect of the invention relates to a method of preserving an ovarian reserve, the method comprising administering to a female subject a composition comprising MIS or an inducible vector that comprises a polynucleotide encoding a recombinant MIS protein.
摘要:
The invention is directed to an adeno-associated virus (AAV) vector which comprises a nucleic acid sequence encoding an antibody that binds to nicotine or a nicotine analog, or an antigen-binding fragment thereof. The invention also is directed to a composition comprising the AAV vector and methods of using the AAV vector to induce an immune response against nicotine in a mammal.
摘要:
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
摘要:
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells, when administered via intravitreal injection, compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
摘要:
Modified capsid proteins, isolated polynucleotides, methods for the preparation of modified capsid proteins, recombinant viral particles, recombinant expression systems for the generation of modified viral particles, and methods of gene editing are provided herein.
摘要:
The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
摘要:
The present invention relates to methods for expressing a polynucleotide of interest in the retina of a subject. In particular, the present inventionrelates to a method for expressing a polynucleotide of interest in the retina of a subject comprising the step consisting of injecting into the vitreous an amount of a vector containing the polynucleotide of interest in combination with an amount of an inhibitor of Dp71 expression.
摘要:
The present invention provides methods and compositions comprising an adeno-associated virus (AAV) capsid protein, comprising one or more amino acids substitutions, wherein the substitutions introduce a new glycan binding site into the AAV capsid protein.
摘要:
The present invention relates to methods for inhibiting myostatin, a regulator of muscle mass, for muscle enhancement (including inducing hypertrophy and/or hyperplasia) as well as improving muscle function (including decreasing atrophy and/or increasing endurance, force and/or strength). The methods involve delivering genes to cells using gene delivery in order to inhibit myostatin. Examples of genes to be delivered are genes encoding' proteins such as Follistatin, Follistatin- related gene-1 (FLRG-I), growth differentiation factor associated protein- 1 (GASP-I) and myostatin precursor propeptide. The genes are delivered using a recombinant Adeno-associated virus (rAAV) lacking rep and cap DNA capable of infecting the cells. Following introduction, the genes are expressed in the cell body of the infected cell and the encoded proteins are secreted systemically. In other methods-of the invention expression of proteins such as activin lib and myostatin is inhibited by oligonucleotide techniques to effect muscle enhancement. All the methods have applications in the treatment of musculoskeletal and neurodegenerative disorders among others, as well as enhancing muscle in livestock.