Hemophilia A: An Ideal Disease for Prenatal Therapy
ABSTRACT Hemophilia A (HA) is the most common inherited coagulation defect. Current state‐of‐the‐art treatment consists of frequent administration of prophylactic infusions of coagulation factor VIII (FVIII) protein or bispecific antibodies that replace the cofactor function of FVIIIa to maintain hemostasis. However, these treatments are far from ideal,
Christopher D. Porada +2 more
wiley +1 more source
Pulmonary infection caused by <i>Tropheryma whipplei</i> in a child before hematopoietic stem cell transplantation: a case report. [PDF]
Zhang K, Zheng J, Wei A, Qin M, Zhu G.
europepmc +1 more source
Diagnosis and Management of Prenatal Hereditary Pyropoikilocytosis
ABSTRACT Hereditary pyropoikilocytosis (HPP) is a severe hemolytic anemia caused by variants in SPTA1, SPTB, and EPB41. These weaken horizontal interactions in the erythrocyte cytoskeleton, causing membrane fragmentation and splenic sequestration. It will readily cause fetal anemia and often hydrops fetalis. Prenatal diagnosis requires first ruling out
Connor Hartzell +6 more
wiley +1 more source
Risk factors associated with chronic cutaneous graft-versus-host disease following hematopoietic stem cell transplantation: a pediatric cohort. [PDF]
Carvajal D +6 more
europepmc +1 more source
ABSTRACT In utero interventions are transformative in addressing genetic and anatomic conditions during fetal development. Next generation sequencing enables early genetic testing, playing a pivotal role in prenatal decision‐making by supporting risk stratification, precise and timely diagnosis, which directly informs eligibility for fetal surgical and
Matthew A. Shear +7 more
wiley +1 more source
A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia. [PDF]
Liu R +29 more
europepmc +1 more source
Attitudes Toward Prenatal Interventions in the Fanconi Anemia Community
ABSTRACT Objective In‐utero cell and gene therapies may offer prenatal treatment options for inherited diseases. Preclinical data suggests in‐utero (IU) hematopoietic stem cell transplantation (HSCT) could prevent Fanconi anemia (FA) related bone marrow failure without genotoxic conditioning or immune suppression.
Tony Lum +4 more
wiley +1 more source
Comprehensive management of hematopoietic stem cell transplantation complications: from infection prevention to immune microenvironment reconstruction. [PDF]
Song Z +6 more
europepmc +1 more source
ABSTRACT Sickle cell disease (SCD) affects millions worldwide but has limited treatment options, most of which carry significant side effects. At present, the only curative treatment for SCD is allogeneic or gene‐modified autologous hematopoietic stem cell (HSC) transplantation (Tx).
Oluwaseun O. Babatunde +4 more
wiley +1 more source

