Mohammed Asmal
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View article: Electrically stimulating sutures: a paradigm shift in wound healing and tissue regeneration
Electrically stimulating sutures: a paradigm shift in wound healing and tissue regeneration Open
View article: The use of lenvatinib and pembrolizumab after platinum-based chemotherapy in advanced endometrial cancer
The use of lenvatinib and pembrolizumab after platinum-based chemotherapy in advanced endometrial cancer Open
View article: Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the <scp>HGB</scp>‐206 study
Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the <span>HGB</span>‐206 study Open
lovo‐cel (bb1111; LentiGlobin for sickle cell disease [SCD]) gene therapy (GT) comprises autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β‐globin gene (β…
View article: Betibeglogene Autotemcel Gene Therapy for Non–β <sup>0</sup> /β <sup>0</sup> Genotype β-Thalassemia
Betibeglogene Autotemcel Gene Therapy for Non–β <sup>0</sup> /β <sup>0</sup> Genotype β-Thalassemia Open
Treatment with beti-cel resulted in a sustained HbAT87Q level and a total hemoglobin level that was high enough to enable transfusion independence in most patients with a non-β0/β0 genotype, including those…
View article: Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia
Wild-type HIV infection after treatment with lentiviral gene therapy for β-thalassemia Open
Betibeglogene autotemcel (beti-cel) gene therapy (GT) for patients with transfusion-dependent β-thalassemia uses autologous CD34+ cells transduced with BB305 lentiviral vector (LVV), which encodes a modified β-globin gene. BB305 LVV also c…
View article: Recurrent Signature Patterns in HIV-1 B Clade Envelope Glycoproteins Associated with either Early or Chronic Infections
Recurrent Signature Patterns in HIV-1 B Clade Envelope Glycoproteins Associated with either Early or Chronic Infections Open
Here we have identified HIV-1 B clade Envelope (Env) amino acid signatures from early in infection that may be favored at transmission, as well as patterns of recurrent mutation in chronic infection that may reflect common pathways of immu…
View article: Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi‐center <scp>HGB</scp>‐206 trial
Safety and feasibility of hematopoietic progenitor stem cell collection by mobilization with plerixafor followed by apheresis vs bone marrow harvest in patients with sickle cell disease in the multi‐center <span>HGB</span>‐206 trial Open
Introducing functional anti-sickling hemoglobin (Hb) via gene transfer into autologous hematopoietic stem and progenitor cells (HSPCs) has a strong therapeutic potential for sickle cell disease (SCD). Typically, HSPCs are obtained by bone …
View article: Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease Open
Key Points Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insert…
View article: PF441 RED BLOOD CELLS PROPERTIES IN PATIENTS WITH SICKLE CELL DISEASE TREATED WITH LENTIGLOBIN GENE THERAPY IN THE HGB‐205 TRIAL
PF441 RED BLOOD CELLS PROPERTIES IN PATIENTS WITH SICKLE CELL DISEASE TREATED WITH LENTIGLOBIN GENE THERAPY IN THE HGB‐205 TRIAL Open
Background: Sickle cell disease (SCD) is one of the most prevalent inherited disorders worldwide. The ex vivo gene therapy phase 1/2 study HGB‐205 conducted in France, evaluates the treatment of SCD and TDT (Transfusion Dependent Thalassem…
View article: 2019 ASPHO ABSTRACTS
2019 ASPHO ABSTRACTS Open
Background: Acute myeloid leukemia (AML) is a heterogeneous disease from morphologic, cytogenetic, immunophenotypic, molecular, and clinical perspectives. AML accounts for15% to 20% of all childhood leukemia. Cytogenetic and molecular data…
View article: Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Outcomes from the Phase 1/2 Northstar and Phase 3 Northstar-2 Studies
Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Outcomes from the Phase 1/2 Northstar and Phase 3 Northstar-2 Studies Open
IntroductionTransfusion-dependent β-thalassemia (TDT) is a severe genetic disease characterized by anemia, iron overload and serious comorbidities for which gene therapy may be an effective treatment option. LentiGlobin gene therapy contai…
View article: Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206
Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206 Open
Introductionβ-globin gene transfer may reduce or eliminate complications in patients with sickle cell disease (SCD). LentiGlobin gene therapy (GT) comprises drug product (DP) made from autologous hematopoietic stem cells (HSCs) transduced …
View article: Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients Open
Novel therapies for sickle cell disease (SCD) based on genetically engineered autologous hematopoietic stem and progenitor cells (HSPCs) are critically dependent on a safe and effective strategy for cell procurement. We sought to assess th…
View article: Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia Open
BACKGROUND:Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transf…
View article: Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in the Northstar Hgb-204 Study
Safety Following Autologous Transplantation with Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in the Northstar Hgb-204 Study Open
Background: Regular life-long red blood cell transfusions and iron chelation are the standard of care for patients with TDT. Allogeneic hematopoietic stem cell transplantation (HSCT) can be curative, but is limited by donor availability an…
View article: Single-Agent Plerixafor Mobilization to Collect Autologous Stem Cells for Use in Gene Therapy for Severe Sickle Cell Disease
Single-Agent Plerixafor Mobilization to Collect Autologous Stem Cells for Use in Gene Therapy for Severe Sickle Cell Disease Open
Background: Patients with severe sickle cell disease (SCD) may benefit from gene therapy using β-globin gene transfer into autologous hematopoietic stem cells (HSCs). Due to risks of leukocytosis, vaso-occlusive crises (VOC), and significa…
View article: Lenti-D Hematopoietic Stem Cell Gene Therapy to Arrest Progression of Cerebral Adrenoleukodystrophy: Interim Results of an International Phase 2/3 Trial
Lenti-D Hematopoietic Stem Cell Gene Therapy to Arrest Progression of Cerebral Adrenoleukodystrophy: Interim Results of an International Phase 2/3 Trial Open
In X-linked adrenoleukodystrophy (ALD), mutations in ABCD1 lead to loss of function of the peroxisomal membrane half-transporter ALD protein and toxic accumulation of very long chain fatty acids. Cerebral ALD (CALD) is characterized by dem…
View article: Infection of rhesus macaques with a pool of simian immunodeficiency virus with the envelope genes from acute HIV-1 infections
Infection of rhesus macaques with a pool of simian immunodeficiency virus with the envelope genes from acute HIV-1 infections Open
View article: Pathogenic infection of Rhesus macaques by an evolving SIV-HIV derived from CCR5-using envelope genes of acute HIV-1 infections
Pathogenic infection of Rhesus macaques by an evolving SIV-HIV derived from CCR5-using envelope genes of acute HIV-1 infections Open
View article: An 86-Year-Old Man With Bilateral Chronic Wounds of the Legs: Figure 1.
An 86-Year-Old Man With Bilateral Chronic Wounds of the Legs: Figure 1. Open
View article: An 86-Year-Old Man With Bilateral Chronic Wounds of the Legs: Figure 1.
An 86-Year-Old Man With Bilateral Chronic Wounds of the Legs: Figure 1. Open
View article: VX-970, selective inhibitor of ataxia telangiectasia and Rad3-related (ATR) protein
VX-970, selective inhibitor of ataxia telangiectasia and Rad3-related (ATR) protein Open