IP Library Granted Patent US 10,500,232
Granted Patent B2
US 10,500,232 · App. 14/913,549 · Granted Dec 10, 2019

Small molecule cellular reprogramming to generate neuronal cells

Inventors: Changsheng Lin (San Francisco, CA); Sheng Ding (Orinda, CA)
Assignee: The J. David Gladstone Ins., a testamentary trust established under the Will of J. David Gladstone
A61K35/30A61K31/165A61K31/167A61K31/192A61K31/196A61K31/198A61K31/222A61K31/352A61K31/4015A61K31/4402A61K31/4406A61K31/4439A61K31/46A61K31/506A61K31/688A61K45/06C12N5/0619C12N2501/01C12N2501/385C12N2501/395C12N2501/415C12N2501/727C12N2501/999C12N2506/1307
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Quick Facts
Patent No.
US 10,500,232
App. No.
14/913,549
Granted
Dec 10, 2019
Kind
B2
Abstract

Compositions and methods are described herein for chemically inducing cells to change their differentiation state and become neuronal cells.

Claims (13)

1. A method of generating a neuronal cell comprising incubating a selected population of non-pluripotent, non-neuronal cells with the composition comprising CHIR99021, an ALK5 and ALK4 inhibitor, a histone deacetylase inhibitor, and a p300 activator, to thereby generate neuronal cells, neuronal progenitor cells, or a combination thereof without de-differentiating the population of non-pluripotent, non-neuronal cells into a population of cells exhibiting pluripotent stem cell characteristics.

2. The method of claim 1 , wherein the selected population of non-pluripotent, non-neuronal cells contacted with the composition comprises a somatic cell, a differentiated cell, a heterogeneous mixture of cells, or a combination thereof.

3. The method of claim 2 , wherein the population of cells comprises cells from the spinal cord or brain.

4. The method of claim 1 , wherein the selected population of non-pluripotent, non-neuronal cells comprises newborn cord blood cells.

5. The method of claim 1 , wherein the composition comprises about 0.01 micromolar to about 20 millimolar CHIR99021, about 0.01 micromolar to about 20 millimolar ALK5 and ALK4 inhibitor, about 0.001 micromolar to about 20 millimolar histone deacetylase inhibitor, and about 0.001 micromolar to about 20 millimolar p300 activator.

6. The method of claim 1 , further comprising administering the neuronal cells, neuronal progenitor cells, or a combination thereof to a subject.

7. The method of claim 6 , wherein the population of non-pluripotent, non-neuronal cells comprises allogenic or autologous cells.

8. The method of claim 6 , wherein the subject suffers or is suspected of suffering from Amyotrophic lateral sclerosis (ALS), Alzheimer's disease, Parkinson's disease, multiple sclerosis, Primary lateral sclerosis (PLS), Progressive bulbar palsy, Pseudobulbar palsy, Primary lateral sclerosis (PLS), Progressive muscular atrophy, Spinal muscular atrophy (SMA), Werdnig-Hoffmann disease, Kugelberg-Welander disease, Fazio-Londe disease, Huntington's disease, Kennedy's disease also known as progressive spinobulbar muscular atrophy, hereditary spastic paraplegia (HSP), congenital SMA with arthrogryposis, Post-polio syndrome (PPS), traumatic spinal cord injury, progressive pseudobulbar palsy, progressive muscular atrophy, stroke, head trauma, spinal cord injury, or a combination thereof.

9. The method of claim 1 , wherein the composition further comprises a PDE4 inhibitor, an Adenylyl cyclase agonist, a retinoic acid receptor γ agonist, a 5-HT3 antagonist, and a metabotropic glutamate (mGlu)receptor agonist.

10. The method of claim 1 , wherein the composition further comprises one or more of the following compounds: a ROCK inhibitor, a neuronal differentiation enhancer, an omega-3 fatty acid, an A3 adenosine receptor agonist or an L-type calcium channel blocker.

11. The method of claim 1 , wherein the composition comprises the following compounds: CHIR99021, SB431542, MS275, N-(4-chloro-3-trifluoromethyl-phenyl)-2-ethoxybenzamide (CTB), Rolipram, Forskolin, CD1530, Tropanyl-3,5-dimethylbenzoate, and (1S,3R)-1-Aminocyclopentane-1,3-dic-arboxylic acid (ACPD).

12. The method of claim 1 , wherein the composition comprises the following compounds: CHIR99021, SB431542, MS275, N-(4-chloro-3-trifluoromethyl-phenyl)-2-ethoxybenzamide (CTB), Rolipram, Forskolin, CD1530, Tropanyl-3,5-dimethylbenzoate, and (1S,3R)-1-Aminocyclopentane-1,3-dicarboxylic acid (ACPD).

13. The method of claim 1 , wherein the composition comprises the following compounds: SB431542, Trichostatin A, Rolipram, CHIR99021, and N-(4-chloro-3-trifluoromethyl-phenyl)-2-ethoxy-6-pentadecyl-benzamide (CTPB).

Assignments (2)
CONFIRMATORY LICENSE Recorded Jul 7, 2016
From: J. DAVID GLADSTONE INSTITUTES
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 039276/0772 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 1, 2016
From: LIN, CHANGSHENG; DING, SHENG
To: THE J. DAVID GLADSTONE INSTITUTES, A TESTAMENTARY TRUST ESTABLISHED UNDER THE WILL OF J. DAVID GLADSTONE
Reel/Frame 037857/0961 →
Continuity (2)
Provisional Application 61870088 · Aug 26, 2013
Related Publication 20160250260A1 · Sep 1, 2016