The present disclosure provides 2-in-1 zinc finger nuclease variants and methods of treating and/or preventing a lysosomal storage disorder using said zinc finger nuclease variants.

The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.

The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.

The present technology generally relates to peptides that bind to the growth hormone receptor (GhR), to peptides that bind to the GhR and have antagonistic activity, and to compositions comprising such peptides.

Compositions and methods for the inhibiting human growth hormone (hGH), and treating or preventing hGH-mediated disorders, using a S1H peptide having the amino acid sequence of [SEQ ID NO: 1], or a variant thereof, are described.

Compositions and methods for the inhibiting human growth hormone (hGH), and treating or preventing hGH-mediated disorders, using a S1H peptide having the amino acid sequence of [SEQ ID NO: 1], or a variant thereof, are described.

A bio-related substance bonded to a branched and degradable polyethylene glycol derivative that is degraded in the cells represented by the following formula (A):

##STR00001##

wherein each symbol is as defined in the present specification, is provided by the present invention.

The present invention discloses an aqueous pharmaceutical composition containing a protein in which serum albumin and growth hormone are linked. The aqueous pharmaceutical composition contains, as an active ingredient, a fusion protein of human serum albumin and human growth hormone; the concentration of the fusion protein is 10-100 mg/mL; the concentration of sucrose is 10-150 mg/mL; the concentration of a nonionic surfactant is 0.15-10 mg/mL; the concentration of a preservative is 0.5-12 mg/mL; the concentration of a buffer is 1-30 mM; and the pH is 5.0-8.0.

The present invention provides a fusion polypeptide comprising a target polypeptide moiety and a self-aggregating peptide moiety, and a method of producing and purifying a target polypeptide by expressing the fusion polypeptide.

The present invention provides a fusion polypeptide comprising a target polypeptide moiety and a self-aggregating peptide moiety, and a method of producing and purifying a target polypeptide by expressing the fusion polypeptide.