Results 41 to 50 of about 20,435 (244)

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +1 more source

Antenatal Screening for Hemoglobinopathies with HPLC [PDF]

, 2018
Introduction: Hemoglobinopathies are the most commonly encountered monogenic disorders of blood in Southeast Asia and Indian subcontinent. Screening of individuals at increased risk of being carriers for thalassemia and hemoglobinopathies, can identify ...
Khonglah, Yookarin, Shah, Naushad, Raphael, Vandana, Nath, Chandan, Singh, A. Santa, Swer, Banylla   +5 more
core   +3 more sources

Perinatal stem-cell and gene therapy for hemoglobinopathies [PDF]

, 2008
Most genetic diseases of the lymphohematopoietic system, including hemoglobinopathies, can now be diagnosed early in gestation. However, as yet, prenatal treatment is not available.
Schoeberlein, Andreina, Wagner, Anna, Surbek, Daniel   +2 more
core   +1 more source

EVOLUTION OF HEMOGLOBINOPATHY PREVENTION IN AFRICA

Mediterranean Journal of Hematology and Infectious Diseases, 2009
Hemoglobinopathies are a group of inherited hemoglobin disorders. Initially described in the subtropical regions, they are now spread all around the world because of migration.
Slaheddine Fattoum
doaj   +1 more source

Presence of new mutations in the TP53 gene in patients with low-risk myelodysplastic syndrome: two case reports

Journal of Medical Case Reports, 2017
Background Myelodysplastic syndromes are heterogeneous disorders. Patients with myelodysplastic syndrome disease often have ineffective hematopoiesis, cytopenias, blood cell dysplasia in one or more cell types, and are at high risk for developing acute ...
Fernando Barroso Duarte, Romélia Pinheiro Gonçalves Lemes, Talyta Ellen de Jesus dos Santos, Maritza Cavalcante Barbosa, João Paulo Leitão de Vasconcelos, Francisco Dário Rocha-Filho, Ilana Zalcberg, Diego Coutinho, Monalisa Feliciano Figueiredo, Luciana Barros Carlos, Paulo Roberto Leitão de Vasconcelos   +10 more
doaj   +1 more source

Hemoglobinopathies in Iran: An Updated Review

International Journal of Hematology-Oncology and Stem Cell Research, 2020
Hemoglobinopathies are the most common single gene disorders (monogenic disorders) in the world population.  Due to specific position of Iran and the presence of multi-ethnic groups in the country, there are many varieties in the molecular genetics and ...
Abolfazl Nasiri, Zohreh Rahimi, Asad Vaisi-Raygani   +2 more
doaj   +1 more source

Plummer–Vinson syndrome in a 10‐year‐old boy from Côte d'Ivoire: An exceptional paediatric case with African context

JPGN Reports, EarlyView.
Abstract Plummer–Vinson syndrome (PVS) is characterised by the triad of dysphagia, iron‐deficiency anaemia, and proximal oesophageal webs. While well described in adults, paediatric cases remain exceptionally rare, particularly in sub‐Saharan Africa.
Paul Mike Tayou Mbobda, Hatrydt Guillaume Dimitri Kouamé, Amadou Ouattara   +2 more
wiley   +1 more source

Venous Oxygenation Measurements in the Upper Arm: Reproducibility and Sensitivity to a Physical Challenge

Magnetic Resonance in Medicine, EarlyView.
ABSTRACT Purpose The feasibility and reproducibility of adapting the T2‐Relaxation‐Under‐Spin‐Tagging (TRUST) MRI sequence for noninvasive measurement of venous oxygenation in the upper arm were investigated, aiming to enable future in vivo validation of T2b‐oxygenation calibration curves in sickle cell disease (SCD).
J. Diogo Fernandes, Koen P. A. Baas, Matthan W. A. Caan, Bart J. Biemond, John C. Wood, Aart J. Nederveen   +5 more
wiley   +1 more source

Prenatal Exome Sequencing Identifies Dual Maternal‐Fetal Diagnosis of HbF Mission Bay, a Novel HBG2 Variant Associated With Methemoglobinemia, Hypoxia and Hemolytic Anemia

Prenatal Diagnosis, EarlyView.
ABSTRACT Prenatal exome sequencing (ES) can establish rare genetic diagnoses in a fetus but may also lead to occult genetic diagnosis in a biological parent. We present a case of dual fetal and maternal diagnosis by prenatal ES, in a fetus with unexplained anemia and in a pregnant patient with sickle cell disease (SCD) and recurrent unexplained hypoxia.
Matthew A. Shear, Billie Lianoglou, Ugur Hodoglugil, W. Patrick Devine, Ashutosh Lal, Juan Gonzalez, Teresa N. Sparks   +6 more
wiley   +1 more source

Iowa Newborn Screening Program Experience with Hemoglobinopathy Screening over the Last Two Decades and Its Increasing Global Relevance

International Journal of Neonatal Screening
Hemoglobinopathies are the commonest monogenic disorder worldwide, with approximately seven percent of the world population being carriers of hemoglobinopathies.
Ryan Jilek, Jennifer Marcy, Carol Johnson, Georgianne Younger, Amy Calhoun, Moon Ley Tung   +5 more
doaj   +1 more source