	US 12,390,491 B2
	Compositions and methods for providing chemoprotective effects
Serhat Gumrukcu, Los Angeles, CA (US)
Assigned to GTECH BIO LLC, Los Angeles, CA (US)
Filed by GTECH BIO LLC, Los Angeles, CA (US)
Filed on Feb. 1, 2021, as Appl. No. 17/164,197.
Claims priority of provisional application 62/968,906, filed on Jan. 31, 2020.
Prior Publication US 2022/0241326 A1, Aug. 4, 2022
Int. Cl. A61K 35/17 (2025.01); A61K 31/015 (2006.01); A61K 31/255 (2006.01); A61K 38/53 (2006.01); A61K 45/06 (2006.01); A61P 39/00 (2006.01); C12N 9/00 (2006.01); C12N 15/113 (2010.01)

CPC A61K 35/17 (2013.01) [A61K 31/015 (2013.01); A61K 31/255 (2013.01); A61K 38/53 (2013.01); A61K 45/06 (2013.01); A61P 39/00 (2018.01); C12N 9/93 (2013.01); C12N 15/1138 (2013.01); C12Y 603/02002 (2013.01); C12N 2310/14 (2013.01); C12N 2310/531 (2013.01)]

11 Claims

1. A method for performing a bone marrow transplant in a patient having HIV, A1AT deficiency, WAS, Hurler Syndrome, Hunter Syndrome, Pompe Disease, Fabry Disease, Mucopolysaccharidoses disorder, MPS 1 H/S (Hurler/Scheie syndrome), MPS I H (Hurler disease), MPS II-(Hunter syndrome), MPS III A, B, C, and D (Sanfillipo syndrome), MPS I S (Scheie syndrome), MPS IV A and B (Morquio syndrome), MPS IX (hyaluronidase deficiency), MPS VII (Sly syndrome), MPS VI (Maroteaux-Lamy syndrome), lysosomal storage diseases, or Childhood cerebral adrenoleukodystrophy (cALD), the method comprising:

administering to the patient a population of busulfan-resistant modified cells and at least one non-myeloablative dose of busulfan,

wherein the population of busulfan-resistant modified cells comprises a heterologous gene encoding glutamate-cysteine ligase (GCL) modifier subunit GCLM, GCLC (GCL catalytic subunit), or GCL (as a dimer or a full enzyme).