Gene therapeutics for treating bone disorders

Abstract

In some aspects, the disclosure relates to compositions and methods for modulating ( e.g., increasing and / or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).

Description

[0001] related application [0002] Pursuant to 35 U.S.C. §119(e), this application claims U.S. Provisional Application No. 62 / 647,595, entitled "Gene Therapy Agents for the Treatment of Bone Disorders," filed March 23, 2018, and February 1, 2019 Priority to U.S. Provisional Application No. 62 / 799,843, entitled "Gene Therapeutic Agents for the Treatment of Bone Disorders," was filed. Background technique [0003] Defects in bone metabolism give rise to a variety of different bone diseases, including those associated with pathological loss or bone mass as well as those associated with pathological increase in bone mass. Effects on bone mass may be systemic or localized. For example, osteoporosis is a disease characterized by decreased bone mass and is a major source of fragility and suffering associated with aging. An estimated 10 million Americans over the age of 50 have osteoporosis, and approximately 1.5 million individuals suffer osteoporosis-related fractures each year, ...

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Problems solved by technology

However, in the context of inflammatory arthritis, anti-sclerostin antibodies have been observed to increase bone tissue destruction and raise stroke risk in the context of TNF-dependent inflammation

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