Abstract: Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.

Abstract: Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.

Abstract: A method for producing polyethylene glycol having a terminal carboxyl group including oxidizing polyethylene glycol represented by formula (I): CH3O(CH2CH2O)n(CH2)m—CHO with potassium peroxymonosulfate in water whose pH is adjusted to be from 6 to 8 with at least one selected from a carbonate, a phosphate, a citrate and an acetate at 5 to 40° C. to produce polyethylene glycol represented by formula (II): CH3O(CH2CH2O)n(CH2)m—COOH, wherein n is a number from 10 to 1,100, and m is 2 or 3.

Abstract: A method for producing polyethylene glycol having a terminal carboxyl group including oxidizing polyethylene glycol represented by formula (I): CH3O(CH2CH2O)n(CH2)m—CHO with potassium peroxymonosulfate in water whose pH is adjusted to be from 6 to 8 with at least one selected from a carbonate, a phosphate, a citrate and an acetate at 5 to 40° C. to produce polyethylene glycol represented by formula (II): CH3O(CH2CH2O)n(CH2)m—COOH, wherein n is a number from 10 to 1,100, and m is 2 or 3.