Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

Inclusion Criteria: * Participants with a bone marrow failure syndrome with an identified genetic cause willing to donate autologous HSPCs for advancing gene therapy * Age ≥ 18 years - 25 years * The following hematological parameters need to be met (regardless of transfusion or growth factor support) * Hb \> 8 g/dL * ANC \> 500/mm3 * Platelet \> 30,000/mm3 * Bone marrow evaluation within the preceding 6 months prior to mobilization and apheresis * Participants should either have a central venous catheter (CVC) in place, be able to undergo apheresis without requiring a CVC, or agree to having a temporary apheresis catheter placed * Karnofsky score \>80 * Negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1/II * Female participants of childbearing age should have a negative serum pregnancy test within one week of beginning Filgrastim and plerixafor administration Exclusion Criteria: * Participant with sickle cell disease * Participant who has had a prior autologous or allogeneic HSCT * Active viral, bacterial, fungal, or parasitic infection * Total bilirubin \>2.5x ULN or transaminases \>5x ULN * Moderate or severe renal failure defined as serum/plasma creatinine \>1.5 mg/dL and an estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m2 based on the CKD-Epi equation or the St. Jude equation * Diagnosis of MDS or other hematologic malignancy * History of malignancy * Known allergy to or contraindication for Filgrastim or plerixafor admini…