Is a
Patent attributes
Patent Applicant
Current Assignee
Patent Jurisdiction
Patent Number
Patent Inventor Names
Michael Heartlein0
Frank DeRosa0
Shrirang Karve0
Date of Patent
June 25, 2024
0Patent Application Number
170024600
Date Filed
August 25, 2020
0Patent Citations
0
...
Patent Primary Examiner
CPC Code
Patent abstract
The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.
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