In the present invention, it has been confirmed that, when an RNA interference-inducing nucleic acid including at least one 8-oxoguanine (o.sup.8G) in 1st to 9th nucleotides from the 5′-end of at least one single strand of a double strand of a nucleic acid, and a modified nucleic acid that specifically binds to microRNA and in which at least one guanine (G) from among the 1st to 9th nucleotides from the 5′-end are modified with 8-oxoguanine (o.sup.8G), are produced and administered to cells or mice, various pathophysiological phenomena are induced.

In addition, the positions where G>T modifications occur have been identified in cDNA produced through the reverse transcription of microRNA in which guanine (G) is oxidatively modified with 8-oxoguanine (o.sup.8G) by oxidative stress in a seed region of microRNA, to confirm the positions where oxidative modification to 8-oxoguanine has occurred.

The present disclosure relates to genetically modified non-human animals that express a fusion protein including B2M and FcRn, and methods of use thereof. In some embodiments, the animals can have a B-NDG background. In some embodiments, the endogenous B2M gene is knocked out in the animals.

Mice, embryos, cells, and tissues having a restricted immunoglobulin heavy chain locus and an ectopic sequence encoding one or more ADAM6 proteins are provided. In various embodiments, mice are described that have humanized endogenous immunoglobulin heavy chain loci and are capable of expressing an ADAM6 protein or ortholog or homolog or functional fragment thereof that is functional in a male mouse. Mice, embryos, cells, and tissues having an immunoglobulin heavy chain locus characterized by a single human V.sub.H gene segment, a plurality of human D.sub.H gene segments and a plurality of human J.sub.H gene segments and capable expressing an ADAM6 protein or ortholog or homolog or functional fragment thereof are also provided.

Compositions and methods for the treatment of muscular dystrophies are provided.

The present invention provides Factor IX fusion proteins with higher specific activity and a longer useful clotting function relative to wild type or non-modified Factor IX protein.

The present invention provides a kidney production method including a step of tissue-specifically removing a metanephric mesenchyme of a metanephros of a non-human animal; a step of transplanting, into the metanephros, a kidney precursor cell derived from a non-human animal which is allogeneic or xenogeneic to the non-human animal; and a step of advancing development of the metanephros, which is a step in which the transplanted kidney precursor cell is differentiated and matured to form a part of the kidney.

The invention discloses methods for the generation of chimaeric human—non-human antibodies and chimaeric antibody chains, antibodies and antibody chains so produced, and derivatives thereof including fully humanised antibodies; compositions comprising said antibodies, antibody chains and derivatives, as well as cells, non-human mammals and vectors, suitable for use in said methods.

Provided are improved compositions and methods for achieving gene therapy in hematopoietic cells and hematopoietic precursor cells, including erythrocytes, erythroid progenitors, and embryonic stem cells. Also provided are improved gene therapy methods for treating hematopoietic-related disorders. Retroviral gene therapy vectors that are optimized for erythroid specific expression and treatment of hemoglobinopathic conditions are disclosed.

Methods of generating spinal cord glutamatergic interneurons (V2a interneurons) from human pluripotent stem cells (hPSCs) are provided. A method of the present disclosure may include culturing a first population of hPSCs in vitro in a neural induction medium that includes: a retinoic acid signaling pathway activator; a sonic hedgehog (Shh) signaling pathway activator; and a Notch signaling pathway inhibitor, wherein the culturing results in generation of a second population of cultured cells containing CHX10+ V2a interneurons. Also provided are non-human animal models that include the hPSC-derived spinal cord glutamatergic interneurons, and methods of producing the non-human animal models.

There is provided a vector for treating retinal degeneration associated with Bardet-Biedl Syndrome (BBS), wherein the vector comprises a promoter operably linked to a BBS1 gene, wherein the promoter is selected from a rhodopsin kinase (RK) promoter, a cytomegalovirus immediate-early (CMV) promoter and a CAG promoter, and wherein the vector is selected from an AAV2/8 vector, an AAV2/7m8 vector and an AAV9 vector. Also disclosed is a pharmaceutical composition comprising the vector, and use of the vector in a method of treating retinal degeneration associated with BBS comprising administering a therapeutically effective amount of the vector to a patient suffering from BBS, wherein the vector is administered directly to the eye of the patient.