Akshay Sharma
- Hematopoietic Stem Cell Transplantation
- Hemoglobinopathies and Related Disorders
- Acute Lymphoblastic Leukemia research
- Acute Myeloid Leukemia Research
- Childhood Cancer Survivors' Quality of Life
- CAR-T cell therapy research
- Prenatal Screening and Diagnostics
- Iron Metabolism and Disorders
- Immune Cell Function and Interaction
- CRISPR and Genetic Engineering
- Virus-based gene therapy research
- Epigenetics and DNA Methylation
- Chronic Myeloid Leukemia Treatments
- Renal Transplantation Outcomes and Treatments
- Neutropenia and Cancer Infections
- Ubiquitin and proteasome pathways
- Parvovirus B19 Infection Studies
- Telomeres, Telomerase, and Senescence
- Chronic Lymphocytic Leukemia Research
- Biomedical Ethics and Regulation
- Palliative Care and End-of-Life Issues
- RNA modifications and cancer
- Blood groups and transfusion
- Bone and Joint Diseases
- Urological Disorders and Treatments
St. Jude Children's Research Hospital
2016-2025
Regional Centre for Biotechnology
2022-2025
Central Drug Research Institute
2019-2024
Children’s Hospital at TriStar Centennial
2021-2024
Morgan Stanley Children's Hospital
2024
The Neurological Institute
2019-2024
Neurological Surgery
2019-2024
Cleveland Clinic
2019-2024
Sawai Man Singh Hospital
2022-2023
Dr. Rajendra Prasad Government Medical College
2020-2023
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression fetal hemoglobin in erythroid cells. We performed electroporation of CD34+ hematopoietic stem progenitor cells obtained from healthy donors, CRISPR-Cas9 targeting the erythroid-specific enhancer. Approximately 80% alleles at this locus were modified, no evidence off-target...
Key Points Cas9 editing of the γ-globin gene promoters in hematopoietic stem cells (HSCs) increases red cell HbF by ≤40%. No deleterious effects on hematopoiesis or off-target mutations were detected 16 weeks after xenotransplantation edited HSCs.
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region
Sickle cell disease is caused by a defect in the β-globin subunit of adult hemoglobin. hemoglobin polymerizes under hypoxic conditions, producing deformed red cells that hemolyze and cause vaso-occlusion results progressive organ damage early death. Elevated fetal levels protect against complications sickle disease. OTQ923, clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9–edited CD34+ hematopoietic stem- progenitor-cell (HSPC) product, has targeted disruption HBG1 HBG2...
As hematopoietic cell transplantation (HCT) and cellular therapy expand to new indications international access improves, the number of HCTs performed annually continues rise. Parallel improvements in HCT techniques supportive care entails more patients surviving long term, creating further emphasis on survivorship needs. Survivors are at risk for developing late complications secondary pretransplantation, peritransplantation, post-transplantation exposures other underlying factors....
Summary Chimeric antigen receptor T‐cell (CAR T‐cell) therapy is associated with significant toxicities secondary to immune activation, including a rare but increasingly recognised severe toxicity resembling haemophagocytic lymphohistiocytosis (carHLH). We report the development of carHLH in 14·8% paediatric patients and young adults treated CD19‐specific CAR carHLH, occurring most commonly those high disease burden. The diagnosis treatment required index suspicion included multidrug...
Abstract The role of haploidentical hematopoietic cell transplantation (HCT) using posttransplant cyclophosphamide (PTCy) for acute lymphoblastic leukemia (ALL) is being defined. We performed a retrospective, multivariable analysis comparing outcomes HCT approaches by donor adults with ALL in remission. primary objective was to compare overall survival (OS) among HCTs PTCy and HLA-matched sibling (MSD), 8/8 unrelated (MUD), 7 /8 HLA-MUD, or umbilical cord blood (UCB) HCT. Comparing MSD HCT,...
Chronic graft- versus -host disease (cGvHD) is a common complication after allogeneic haematopoietic stem cell transplantation, characterised by broad spectrum that can affect virtually any organ. Although pulmonary cGvHD less manifestation, it of great concern due to its severity and poor prognosis. Optimal management patients with complicated no standardised approach available. The purpose this joint European Respiratory Society (ERS) for Blood Marrow Transplantation task force was develop...
Allogeneic hematopoietic cell transplantation (HCT) can be complicated by life-threatening organ toxicity and infection necessitating intensive care. Epidemiologic data have been limited single-center studies, poor database granularity, a lack of long-term survivors. To identify contemporary trends in care unit (ICU) use outcomes, we merged from the Center for International Blood Marrow Transplant Research Virtual Pediatric Systems databases. We identified 6995 pediatric patients with HCT...
Abstract There has been an increase in volume as well improvement overall survival (OS) after hematopoietic cell transplantation (HCT) for hematologic disorders. It is unknown if these changes have affected racial/ethnic minorities equally. In this observational study from the Center International Blood and Marrow Transplant Research of 79 904 autologous (auto) 65 662 allogeneic (allo) HCTs, we examined rates change autoHCT alloHCT over time trends OS 4 groups: non-Hispanic Whites (NHWs),...
Post-transplant cyclophosphamide (PTCy) has significantly increased the successful use of haploidentical donors with a relatively low incidence graft-versus-host disease (GVHD). Given its increasing use, we sought to determine risk factors for GVHD after hematopoietic cell transplantation (haplo-HCT) using PTCy. Data from Center International Blood and Marrow Transplant Research on adult patients acute myeloid leukemia, lymphoblastic myelodysplastic syndrome, or chronic leukemia who...
Abstract Optimal post-remission therapy for adolescents and young adults (AYAs) with Ph-negative acute lymphoblastic leukemia (ALL) in first complete remission (CR1) is not established. We compared overall survival (OS), disease-free (DFS), relapse, non-relapse mortality (NRM) patients receiving on CALGB 10403 to a cohort undergoing myeloablative (MA) allogeneic hematopoietic cell transplantation (HCT) CR1. In univariate analysis, OS was superior chemotherapy MA HCT (3-year 77% vs. 53%, P...
Abstract Food safety and foodborne diseases are significant global public health concerns. Meat poultry carcasses can be contaminated by pathogens like E. coli salmonella, contact with animal fecal matter ingesta during slaughter processing. Since host these pathogens, detection, excision of regions on meat surfaces is crucial. Fluorescence imaging has proven its potential for the detection residue but requires expertise to interpret. In order used cutters without special training, automated...
Hematopoietic stem cell transplantation (HSCT) is curative for many non-malignant disorders. As HSCT and supportive care technologies improve, this life-saving treatment may be offered to more patients. With the development of new preparative regimens, expanded alternative donor availability, graft manipulation techniques, there are options when choosing best regimen Herein authors review transplant considerations, goals, conditioning choice, strategies patients with disorders undergoing HSCT.