Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
US9770471B2 · kind B2 · utility
Assignees
Inventors
Key dates
| Filing date | Aug 17, 2012 |
| Grant date | Sep 26, 2017 |
| Priority date | — |
| Expiry date | Oct 21, 2033 |
Classification
- Technology area (CPC Y)Emerging Cross-Sectional Technologies
- CPC primaryY02A50/30
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention provides methods of transdifferentiation of somatic cells, for example, directly converting a somatic cell of a first cell type, e.g., a fibroblast into a somatic cell of a second cell type, are described herein. In particular, the present invention generally relates to methods for converting a somatic cell, e.g., a fibroblast into a motor neuron, e.g., an induced motor neuron (iMN) with characteristics of a typical motor neuron. The present invention also relates to an isolated population comprising induced motor neurons (iMNs), compositions and their use in the treatment of motor neuron diseases such as ALS and SMA. In particular, the present invention relates to direct conversion of a somatic cell to an induced motor neuron (iMN) having motor neuron characteristics by increasing the protein expression of at least three motor-neuron inducing (MN-inducing) factors selected from Lhx3, Ascl1, Brn2, Myt1l, Isl1, Hb9, Ngn2 or NeuroD1 in a somatic cell, e.g., a fibroblast to convert the fibroblast to an induced motor neuron (iMN) which exhibits at least two characteristics of an endogenous motor neuron.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.