Towards a Cure for Diamond–Blackfan Anemia: Views on Gene Therapy
M Vale, J Prochazka, R Sedlacek - Cells, 2024 - mdpi.com
Diamond–Blackfan anemia (DBA) is a rare genetic disorder affecting the bone marrow's
ability to produce red blood cells, leading to severe anemia and various physical …
ability to produce red blood cells, leading to severe anemia and various physical …
Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment
JL Vives Corrons - Journal of Clinical Medicine, 2024 - mdpi.com
Background—This review provides a comprehensive overview of rare anemias,
emphasizing their hereditary and acquired causes, diagnostic advancements, and evolving …
emphasizing their hereditary and acquired causes, diagnostic advancements, and evolving …
The CRISPR-Cas System and Clinical Applications of CRISPR-Based Gene Editing in Hematology with a Focus on Inherited Germline Predisposition to Hematologic …
R Kansal - Genes, 2024 - mdpi.com
Clustered regularly interspaced short palindromic repeats (CRISPR)-based gene editing
has begun to transform the treatment landscape of genetic diseases. The history of the …
has begun to transform the treatment landscape of genetic diseases. The history of the …
Mechanistic and Biological Perspectives on Erythropoiesis
EH Bresnick, T Papayannopoulou… - Experimental …, 2024 - exphem.org
Although red blood cells (RBC) have been studied for decades, investigations into
mechanistic and biological aspects of these essential cells continue to yield transformative …
mechanistic and biological aspects of these essential cells continue to yield transformative …
CRISPR RNA-Guided Gene Editing and its Clinical Research Applications in Hematology with Focus on Inherited Germline Predisposition to Hematologic …
R Kansal - 2024 - preprints.org
Clustered regularly interspaced short palindromic repeats (CRISPR) based gene-editing
has begun to transform the treatment landscape of genetic diseases. The history of the …
has begun to transform the treatment landscape of genetic diseases. The history of the …
[HTML][HTML] Anemia di Diamond-Blackfan La malattia si manifesta con grave anemia dalla nascita o dai primi mesi di vita cui si possono associare malformazioni …
N Tovaglieri, E Altieri - malattierare.eu
tab1 L'anemia di Diamond-Blackfan (DBA) è una rara aplasia congenita della serie eritroide
che provoca una grave anemia normocromica-macrocitica. Descritta per la prima volta nel …
che provoca una grave anemia normocromica-macrocitica. Descritta per la prima volta nel …