Described herein are novel divalent nucleobases that each bind two nucleic acid strands, matched or mismatched when incorporated into a nucleic acid or nucleic acid analog backbone (a genetic recognition reagent, or genetic recognition reagent). In one embodiment, the genetic recognition reagent is a peptide nucleic acid (PNA) or gamma PNA (γPNA) oligomer. Uses of the divalent nucleobases and monomers and genetic recognition reagents containing the divalent nucleobases also are provided.

Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.

Disclosed herein, interalia, are compounds, compositions, and methods of using the same for the sequencing of a nucleic acid.

The invention provides a method for synthesizing a DNA sequence comprising repeat units, including designing and synthesizing an extension primer and a blocking primer based on the repeat unit, performing a PCR amplification reaction by using the repeat unit (as an amplification template), the extension primer, and the blocking primer in a PCR reaction system, to obtain the DNA sequence comprising repeat units. The invention also provides a kit for this method. The method of the invention has the characteristics such as controllable copy number for repeat synthesis, simple synthesis steps, and low cost, and is very suitable for high-throughput production in industry.

Methods of labelling one or more subcellular components (e.g., an organelle and/or subcellular region) in vivo are provided. Methods of labelling a protein in vivo are provided. Methods of determining a nucleic acid sequence in situ are also provided.

Methods of labelling one or more subcellular components (e.g., an organelle and/or subcellular region) in vivo are provided. Methods of labelling a protein in vivo are provided. Methods of determining a nucleic acid sequence in situ are also provided.

A microfluidic valve includes a carrier layer and a flexible membrane layer arranged on a surface of the carrier layer. The surface of the carrier layer has a valve chamber in the form of a spherical cap and a membrane formed by the flexible membrane layer covers at least the valve chamber. A plurality of microfluidic channels opening into the valve chamber are formed in the surface of the carrier layer. Moreover, an inflow channel and an outflow channel are connected to one another by a microfluidic connection channel. The connection channel and the valve chamber are positioned relative to each other in such a way that in the closed state of the membrane, a fluid can flow from the inflow channel via the connection channel into the outflow channel to bridge the valve chamber, while the at least one supply channel is closed by the membrane.

A microfluidic valve includes a carrier layer and a flexible membrane layer arranged on a surface of the carrier layer. The surface of the carrier layer has a valve chamber in the form of a spherical cap and a membrane formed by the flexible membrane layer covers at least the valve chamber. A plurality of microfluidic channels opening into the valve chamber are formed in the surface of the carrier layer. Moreover, an inflow channel and an outflow channel are connected to one another by a microfluidic connection channel. The connection channel and the valve chamber are positioned relative to each other in such a way that in the closed state of the membrane, a fluid can flow from the inflow channel via the connection channel into the outflow channel to bridge the valve chamber, while the at least one supply channel is closed by the membrane.

The present invention includes synthesis of polyethyleneimine800-EpoxyC8-22 (PEI800-C8-22) lipids, e.g., Polyethyleneimine800-EpoxyC16 (PEI800-C16), PEI12C16, PEI8C16, and PEI4C16 lipids, compositions and methods for transfecting primary leukocytes, myeloid cells, lymphoid cells, monocytes, macrophages and dendritic cells (DC) comprising a transfection complex comprising: one or more nanoparticles; and Polyethyleneimine800-EpoxyC16 (PEI800-C16), PEI12C16, PEI8C16, and PEI4C16 lipids complexed with one or more nucleic acids, such as, e.g., DNA, RNA, nucleic acid vectors, shRNA, miRNA, and RNAi on or about the nanoparticles.

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.