Correction of genetic defects
AssigneesWhitehead Institute for Biomedical Research, The General Hospital Corporation
InventorsWilliam Rideout, Konrad Hochedlinger, Michael Kyba, Rita Perlingeiro, George Q. Daley, Rudolf Jaenisch
Life of this patent
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Abstract
A method of correcting of treating a genetic disorder by combining therapeutic cloning and gene therapy.
Claims23
A method of correcting a genetic defect in an individual in need thereof, comprising administering to the individual a therapeutically effective amount of repaired ntES cells and/or repaired differentiated progenitor or precursor cells derived from repaired ntES cells, wherein in the repaired ntES cells, the genetic defective has been corrected.
A method of treating a genetic disorder in an individual in need thereof, comprising administering to the individual a therapeutically effective amount of repaired ntES cells and/or repaired differentiated progenitor or precursor cells derived from repaired ntES cells, wherein in the repaired cells, a defect in a gene or genes that causes or is associated with the genetic disorder has been corrected.
Repaired ntES cells.
A method of producing repaired ntES cells, comprising:
- (a) introducing nuclei from a somatic cell into enucleated oocytes, wherein the somatic cell comprises DNA comprising a genetic defect;
- (b) maintaining the product of (a) under conditions appropriate for blastocyst formation, thereby producing blastocysts comprising DNA from the somatic cell;
- (c) obtaining embryonic stem cells from blastocysts produced in (b), wherein the embryonic stem cells are referred to as ntES cells and comprise DNA comprising the genetic defect;
- and
- (d) correcting the genetic defect in the ntES cells, thereby producing repaired ntES cells.
In this method, the genetic defect can be corrected in ntES cells by a recombinant nuclei acid method (e.g., homologous recombination). In this embodiment, homologous recombination occurs between (a) DNA in ntES cells which comprises the genetic defect to be corrected and (b) DNA that (i) is introduced into the ntES cells;
- (ii) comprises DNA that, when introduced into DNA in the ntES cells corrects the genetic defect;
- and (iii) undergoes homologous recombination with DNA in the ntES cells in such a manner that the genetic defect is corrected, thereby correcting the genetic defect in the ntES cells and resulting in production of repaired ntES cells.
The genetic defect corrected can be, for example, a genetic defect that causes an immune system disorder;
- a genetic defect that causes a neurological disorder;
- a genetic defect that causes a cardiac disorder;
- a genetic defect that causes a circulatory disorder or a genetic defect that causes a respiratory disorder.
Dependencies are inferred from the claim text.