Acute Promyelocytic Leukemia (APL)
Acute promyelocytic leukemia (APL) represents a unique subtype of AML characterized by the PML-RARA fusion gene. You need specialized drug and therapy development services to improve and accelerate APL research. Our company is at the forefront of providing comprehensive APL drug and therapy development services.
Introduction to APL
Acute promyelocytic leukemia (APL) is a rare and aggressive form of AML, comprising approximately 10% of all AML cases. It is characterized by the fusion of the promyelocytic leukemia retinoic acid receptor alpha (PML-RARA) gene due to the t(15;17) chromosomal translocation. This fusion gene disrupts the normal differentiation process of myeloid cells, leading to the accumulation of immature promyelocytes.
Pathogenesis of APL
The pathogenesis of APL is primarily driven by the t(15;17) chromosomal translocation, which results in the fusion of the PML and RARA genes. This fusion gene, PML-RARA, disrupts the normal function of both genes and leads to the arrest of myeloid cell differentiation at the promyelocyte stage. The accumulation of these immature cells contributes to the development of APL. Individuals with APL typically present with symptoms such as bleeding, fatigue, and an increased risk of infections due to impaired normal blood cell production.
Diagnostics Development of APL
The world of diagnostic development for APL has witnessed remarkable progress in recent years, propelling the field into a new era of precision medicine. The groundbreaking identification of the t(15;17) chromosomal translocation and the consequential fusion of the PML-RARA genes has triggered a seismic shift in the way APL is diagnosed and treated. Polymerase chain reaction (PCR) and fluorescence in situ hybridization (FISH) have emerged as the vanguards of diagnostic methodologies, exhibiting unparalleled sensitivity and specificity in detecting the PML-RARA fusion gene.
Fig.1 Evolution of therapy in APL. (Yilmaz, Musa, et al., 2021)Therapy Development of APL
Small Molecule Drugs
Small molecule drugs, such as all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), have shown remarkable success in the therapy of APL. ATRA promotes the differentiation of APL cells by targeting the PML-RARA fusion protein, while ATO induces apoptosis and degradation of the fusion protein.
Gene Therapies
Gene therapies offer a potential long-term solution for APL. These approaches involve modifying or correcting the genetic abnormalities responsible for APL development. For instance, gene editing technologies, such as CRISPR-Cas9, hold promise in selectively targeting and disrupting the PML-RARA fusion gene.
Our Services
In the ever-evolving landscape of diagnostics development for APL, our company stands as an indomitable force at the forefront of innovation. At the nucleus of our diagnostic arsenal lie the cutting-edge PCR and FISH assays, which serve as the bedrock of our diagnostic prowess. These outstanding technologies combine sensitivity and specificity to enable us to conduct APL studies with extremely high precision.
Platforms of APL Therapy Development
We offer a full range of research services to help you develop therapies and accelerate preclinical studies. Our comprehensive suite of services encompasses rare disease model studies, drug safety evaluation, and pharmacokinetic analysis, all meticulously designed to catalyze the development of APL drugs.
Animal Models of APL
- Transgenic models (PML-RARalpha)
- Bone marrow transduction models
If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
- Yilmaz, Musa, Hagop Kantarjian, and Farhad Ravandi. "Acute promyelocytic leukemia current treatment algorithms." Blood cancer journal 11.6 (2021): 123.
- Jimenez, Joaquin J., et al. "Acute promyelocytic leukemia (APL): a review of the literature." Oncotarget 11.11 (2020): 992.
- Sanz, Miguel A., and Eva Barragán. "History of acute promyelocytic leukemia." Clinical Hematology International 3.4 (2021): 142.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.