Stem Cell Mobilization and Apheresis for Life-threatening Blood Disorders

The purpose of this study is to investigate mobilization and collection of HSPCs in patients with bone marrow failure syndromes (BMFS) using granulocyte-colony stimulating factor (otherwise known as Filgrastim) with plerixafor to demonstrate safety and feasibility of collecting HSPCs to advance gene therapy. Primary objective: \- To characterize the safety of Filgrastim plus plerixafor in participants with bone marrow failure syndromes as determined by the incidence of adverse events (AEs). Secondary Objectives: * To characterize the feasibility of HSPC mobilization using Filgrastim plus plerixafor as determined by peripheral blood CD34+ counts. * To measure the mobilization effects of Filgrastim plus plerixafor in the peripheral blood in participants as determined by peak peripheral blood CD34+ counts. * To estimate efficacy of Filgrastim plus plerixafor for HSPC mobilization and apheresis collection in participants as determined by the yield of CD34+ cells (CD34+ cells/kg).

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 administration, or medications routinely administered during apheresis * Splenomegaly (size greater than upper limit of normal on examination) * Any disease or concomitant process that is not compatible with the study as per investigator opinion * Concomitant treatment with alternative investigational agent or participation in another clinical trial with an investigational drug within 5 half-lives of the investigational agent * Unwillingness to use a highly effective method of contraception for 1 month after plerixafor or GCSF * Pregnancy * Inability or unwillingness of research participant to give written informed consent.