Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3 UTR, and a prompter.

Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3 UTR, and a promoter.

A method of detecting Polycomb Repressive Complex (PRC) activity in a cell providing a cell with a DNA having a protein binding site and at least one reporter gene expression site is operatively connected to the protein binding site, and with a DNA containing a recombinant gene of a binding protein, the binding protein being capable of binding to the protein binding site, wherein the binding protein is fused to a member of the PRC, the method including the step of expressing the recombinant gene, letting the fused binding protein bind to the protein binding site and detecting at least one reporter gene expression.

A method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first, second or third vector, wherein the first vector comprises a promoter enhancer, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase and an albumin 3UTR and lacks an HGH intron, wherein the second vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site and an albumin 3UTR and lacks a promoter enhancer, wherein the third vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site, an albumin 3UTR and a promoter enhancer and observing the patient's insulin levels, wherein the patient's insulin levels are controlled.

Provided is a system for regulating expression of a gene of interest in a target cell, including a recombinant first RNA molecule with (i) a coding sequence for a translation-suppressor protein and (ii) a first microRNA (miR) recognition element in its 3 UTR, wherein the first miR recognition element recognizes a first miR and binding of a first miR to the first miR recognition element reduces translation of the translation suppressor, and a recombinant second RNA molecule, with (i) a coding sequence for the gene of interest, (ii) a recognition sequence for the translation-suppressor, wherein binding of the translation-suppressor to the recognition sequence for the translation-suppressor reduces translation of the gene of interest, and, optionally, (iii) a second miR recognition element in its 3 UTR, wherein the second miR recognition element recognizes one or more second miR and binding of one or more of the one or more second miR to the second miR recognition element reduces translation of the gene of interest. Also provided are methods of using the system.

UO.sub.2F.sub.2 biosensors, and related U-sensing and/or F-sensing genetic molecular components, genetic circuits, compositions, methods and systems are described, which in several embodiments can be used to detect and/or neutralize uranium and in particular bioavailable UO.sub.2F.sub.2.

There is provided a retroviral RNA vector comprising a 5 cap, a transgene, a 3 long terminal repeat (LTR) and an RNA packaging sequence, wherein translation of the transgene is initiated at the 5 end of the transgene in a cap-dependent manner, and wherein the 3 LTR and the RNA packaging sequence are located 3 of the transgene. Also provided is a nucleotide sequence encoding a vector genome. In addition, there is provided a host cell, a virion and a pharmaceutical composition comprising the vector or nucleotide sequence, and the use of the vector in delivering a transgene to a cell or subject.

Systems, methods, and kits for enhancing mRNA translation are disclosed. Some embodiments describe expression constructs for producing a peptide and include a translational enhancer. Additional embodiments describe methods for producing a peptide using a construct including a translational enhancer. Certain embodiments further enhance mRNA stability.

The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3-untranslated region element (3-UTR element) and/or at least one 5-untranslated region element (5-UTR element), wherein the at least one 3-UTR element and/or the at least one 5-UTR element prolongs and/or increases protein production from said artificial nucleic acid molecule and wherein the at least one 3-UTR element and/or the at least one 5-UTR element is derived from a stable mRNA. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, methods for identifying a 3-UTR element and/or a 5-UTR derived from a stable mRNA element are disclosed.

Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.