Provided is a promoter having high activity in an activated T-cell. The promoter comprises, from 5′-end to 3′-end, a CMV enhancer, an IFNγ promoter, and a long terminal repeat sequence from human T-cell leukemia virus that are connected in sequence. The promoter exhibits greater activity in an activated immune cell than the existing promoters and is low in activity or inactive in other non-immune cells.

Disclosed is an erythroid-specific human β-globin gene promoter and a human β-globin-expressing recombinant sequence, and use thereof, which belongs to the technical field of genetic engineering. An erythroid-specific human β-globin gene promoter with a nucleotide sequence set forth in SEQ ID NO: 1 is provided in the present disclosure, which may achieve high-efficiency and erythroid-specific initiation of the expression of a functional gene. A recombinant sequence specifically expressing human β-globin in erythroid cells is also provided in the present disclosure, which is human β-globin locus control region (LCR) HS 3-1+β-globin gene promoter+β-globin gene+β-globin gene enhancer.

Disclosed herein are novel variants of KCVN2 and their use, for example, in methods of treating a subject with a retinal disorder, such as CDSRR.

The present invention provides an isolated nucleic acid molecule comprising, or consisting of, the nucleic acid sequence of SEQ ID NO:1 or a nucleic acid sequence of at least 1000 bp having at least 80% identity to said sequence of SEQ ID NO:1, wherein said isolated nucleic acid molecule specifically leads to the expression in cells of the retinal pigment epithelium of a gene when operatively linked to a nucleic acid sequence coding for said gene.

A nerve in a mammal is optogenetically transduced, wherein the nerve is susceptible to stimulus by selective application of transdermal light, and a light source is applied to dermis of the mammal at or proximate to the optogenetically transduced nerve, to thereby stimulate the nerve. A wearable device for optogenetic motor control and sensation restoration of a mammal includes a wearable support, a power source at the wearable support, a controller at the wearable support and in electrical communication with a power source, and a transdermal light source coupled to the controller.

The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 capsid and a codon-optimized sequence encoding a human phenylalanine hydroxylase (PAH) enzyme. The disclosure also provides a method of treating a subject having phenylketonuria (PKU), comprising administering to the subject in need thereof a recombinant adeno-associated vims (rAAV) vector comprising an AAV8 capsid, and a promoter operably linked to a nucleic acid sequence that encodes PAH, and wherein administering results in a decrease in phenylalanine level in the subject.

Methods are disclosed for polarizing macrophages to become M2 macrophages. Methods also are disclosed for treating type 1 diabetes in a subject. These methods include administering to the subject a vector comprising a macrophage specific promoter operably linked to a nucleic acid molecule encoding TNF-alpha-induced protein 8-like 2 (TIPE2) protein. In some embodiments, the vector is administered locally to a pancreas of the subject. In further embodiments, compositions are disclosed including a) a vector comprising a macrophage specific promoter operably linked to a nucleic acid molecule encoding TNF-alpha-induced protein 8-like 2 (TIPE2) protein; b) a buffer; and c) a contrast dye for endoscopic retrograde cholangiopancreatography.

The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscles including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.

The invention provides nucleic acids and nucleic acid expression vectors containing optimized mGluR6 promoters for expression of transgenes in the retina. The compositions and methods of the invention are useful for expression of gene products to preserve, improve, or restore phototransduction or vision.

The present invention provides methods for treating an individual having solid or lymphatic tumor comprising locally administering to the site of the tumor an oncolytic virus, and systemically administering an immunomodulator (including a combination of immunomodulators). The methods may further comprise local administration to the site of the tumor a second immunomodulator (including a combination of immunomodulators). Also provided are compositions and kits for the cancer therapy methods.