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AssigneeVitro Biopharma, Inc.
InventorsJames R. MUSICK, Tiana States, Caroline MOSESSIAN
Estimating the term…
Provided herein are gene and cell therapy-based compositions and methods of treating Pitt-Hopkins Syndrome (PTHS). The methods of treating PTHS utilize compositions that contain an effective amount of umbilical cord-derived mesenchymal stem cells (UC-MSCs), isolated exosomes derived from the UC-MSCs, cell culture medium containing the UC-MSC's secretome (e.g., UC-MSC secretome-conditioned cell culture medium), and cell culture medium containing the UC-SLMSC's secretome but devoid of exosomes (e.g., exosome-depleted UC-MSC-conditioned cell culture medium). The compositions described herein additionally contain Wnt pathway activators to increase transcription factor 4 (TCF4 expression) in MSCS.
A method of treating a subject with Pitt-Hopkins Syndrome (PTHS) comprising administering to the subject an effective amount of a composition comprising:
A pharmaceutical composition comprising:
A pharmaceutical composition for use in treating PTHS comprising:
Dependencies are inferred from the claim text.