The major β‐haemoglobinopathies, sickle cell disease and β‐thalassaemia, represent the
most common monogenic disorders worldwide and a steadily increasing global disease …

The clustered regularly interspaced short palindromic repeats (CRISPR) system of genome
editing represents a major technological advance spanning all areas of genetics and …

CD33 has long been pursued as immunotherapeutic target in acute myeloid leukemia
(AML)[1, 2]. Improved survival with gemtuzumab ozogamicin (GO) validates this approach …

The identification of new and even more precise technologies for modifying and
manipulating the genome has been a challenge since the discovery of the DNA double …

Clustered regularly interspaced short palindromic repeats (CRISPR) has revolutionized
many research areas and has rapidly become the gold standard in genome editing by …

Hematopoiesis is one of the best studied adult stem-cell systems, with a differentiation
hierarchy progressing from immature hematopoietic stem cells to over 10 distinct mature cell …

Background Multiple Myeloma (MM) is a heterogeneous, hematological neoplasm that
accounts 2% of all cancers. Although, autologous stem cell transplantation and …

Hematological malignancies (HM) are a group of neoplastic diseases that are usually
heterogenous in nature due to the complex underlying genetic aberrations in which …

Gene therapy has been a long lasting goal for scientists, and there are many optimal
methods and tools to correct disease-causing mutations in humans. Recently, the Clustered …

Haematopoietic stem cells (HSCs) have the potential for lifetime production of blood and
immune cells. The introduction of transgenes into HSCs is important for basic research, as …