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: The invention provides methods for treating neutropenia in a subject in need thereof comprising providing a composition comprising a retinoid agonist and administering an effective amount of the composition to the subject to treat neutropenia, thereby treating neutropenia to the subject in need thereof.

Abstract: The invention relates methods of using a retinoid agonist and a G-CSF or an analog thereof to treat, prevent, reduce the likelihood of having, reduce the severity of and/or slow the progression of a condition in a subject. The retinoid agonist and the G-CSF or the analog thereof may be provided in a single composition or in separate compositions. A therapeutically effective amount of the retinoid agonist and the G-CSF or the analog thereof may be administered to the subject concurrently or sequentially. Conditions treatable with the methods and compositions include but are not limited to various forms of neutropenia and microbial infections. The invention also relates to methods for determining the efficacy of the treatments described herein.

Abstract: The invention relates methods of using a retinoid agonist and a G-CSF or an analog thereof to treat, prevent, reduce the likelihood of having, reduce the severity of and/or slow the progression of a condition in a subject. The retinoid agonist and the G-CSF or the analog thereof may be provided in a single composition or in separate compositions. A therapeutically effective amount of the retinoid agonist and the G-CSF or the analog thereof may be administered to the subject concurrently or sequentially. Conditions treatable with the methods and compositions include but are not limited to various forms of neutropenia and microbial infections. The invention also relates to methods for determining the efficacy of the treatments described herein.

Abstract: The invention provides methods for treating neutropenia in a subject in need thereof comprising providing a composition comprising a retinoid agonist and administering an effective amount of the composition to the subject to treat neutropenia, thereby treating neutropenia to the subject in need thereof.