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Comprehensive view on chemotherapy-free management of acute myeloid leukemia by using venetoclax in combination with targeted and/or immune therapies.

A hallmark of cancer biology is resistance to apoptosis. BCL-2 is an anti-apoptotic molecule that is being overexpressed in several myeloid … eventually to its first US FDA approvals in 2018. As the modern approach to treating hematological malignancies are the chemotherapy-free regimen, in the current manuscript, we provide a comprehensive view on all available therapies that are considered to be chemotherapy-free, with a special emphasis on acute myeloid leukemia (AML), where phase I-III …

Application of the CRISPR/Cas9-based gene editing technique in basic research, diagnosis, and therapy of cancer.

The 2020 Nobel Prize in Chemistry was awarded to Emmanuelle Charpentier and Jennifer Doudna for the development of the Clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease9 (CRISPR/Cas9) gene editing technology that provided new tools for precise gene editing. It is possible to target any genomic locus virtually using only a complex nuclease protein with short RNA as a site-specific endonuclease. Since cancer is caused by genomic changes in tumor cells, CRISPR/Cas9 can be used in the field of cancer research to edit genomes for exploration of the mechanisms of t

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… In 2013, Cong and Mali et al. applied the type II Cas system to the cutting of DNA in mammalian cells [ 14 , 25 ], which paved the way for the application of the CRISPR/Cas9 system in genome modification. … Mali P Yang L Esvelt KM Aach J Guell M DiCarlo JE RNA-guided human genome engineering via Cas9 Science. 2013 339 823 826 10.1126/science.1232033 23287722 PMC3712628 26. …

Adjuvant breast cancer treatments cardiotoxicity and modern methods of detection and prevention of cardiac complications.

The most common cancer diagnosis in female population is breast cancer, which affects every year about 2.0 million women worldwide. In recent years, significant progress has been made in oncological therapy, in systemic treatment, and in radiotherapy of breast cancer. Unfortunately, the improvement in the effectiveness of oncological treatment and prolonging patients' life span is associated with more frequent occurrence of organ complications, which are side effects of this treatment. Current recommendations suggest a periodic monitoring of the cardiovascular system in course of oncological t

The 4th Global Congress on Sickle Cell Disease Abstract Book 2022

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… The authors do not declare any conflict of interest PI-09: LESSONS LEARNED FROM ONE DECADE EXPERIENCE OF THE « CENTRE DE RECHERCHE ET DE LUTTE CONTRE LA DRÉPANOCYTOSE (CRLD) » IN BAMAKO, MALI. … In Mali, the birth rate was estimated at 714,000 per year and the incidence of sickle cell anemia (homozygous SS + S/β-thalassemia + SC), at 0.8% of live births. …

Mapping Cancer in Africa: A Comprehensive and Comparable Characterization of 34 Cancer Types Using Estimates From GLOBOCAN 2020.

ObjectiveCancer incidence and mortality rates in Africa are increasing, yet their geographic distribution and determinants are incompletely characterized. The present study aims to establish the spatial epidemiology of cancer burden in Africa and delineate the association between cancer burden and the country-level socioeconomic status. The study also examines the forecasts of the cancer burden for 2040 and evaluates infrastructure availability across all African countries.MethodsThe estimates of age, sex, and country-specific incidence and mortality of 34 neoplasms in 54 African countries, we

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… 3,552 [2,842–4,439] | 119.3 | 90.5 | 2,603 [2,114–3,205] | 0.73 Libya | 7,661 [7,063–8,309] | 132.2 | 88.2 | 4,750 [4,381–5,150] | 0.62 Madagascar | 20,681 [18,358–23,298] | 124.3 | 87.7 | 13,837 [12,282–15,588] | 0.67 Malawi | 17,936 [16,569–19,416] | 154.2 | 113.4 | 12,454 [11,444–13,553] | 0.69 Mali …

Gene Therapy for Pancreatic Diseases: Current Status.

The pancreas is a key organ involved in digestion and endocrine functions in the body. The major diseases of the pancreas include pancreatitis, pancreatic cancer, cystic diseases, pancreatic divisum, islet cell tumors, endocrine tumors, diabetes mellitus, and pancreatic pain induced by these diseases. While various therapeutic methodologies have been established to date, however, the improvement of conventional treatments and establishment of novel therapies are essential to improve the efficacy. For example, conventional therapeutic options, including chemotherapy, are not effective against p

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… Mali P. Wu E.Y. Ng A.H. Zhu K. Wagers A.J. Church G.M. A multifunctional AAV-CRISPR-Cas9 and its host response Nat. Methods 2016 13 868 874 10.1038/nmeth.3993 27595405 PMC5374744 61. Chiou S.H. Winters I.P. Wang J. Naranjo S. Dudgeon C. Tamburini F.B. Brady J.J. Yang D. Grüner B.M. Chuang C.H. …

Role of exosomes in pancreatic cancer.

Pancreatic cancer is one of the most lethal malignancies. Exosomes, which are released by multiple cell types, such as cancer cells, contain functional biomolecules (including proteins, nucleic acids and lipids) that can be horizontally delivered to recipient cells. Exosomes act as the most prominent mediator of intercellular communication and can regulate, instruct and re-educate their surrounding microenvironment and target specific organs. The present review performed an extensive search of multiple databases from 2005 to April 23 2017, for eligible literature relating to exosomes and their…

Surgical versus non-surgical treatment for vertebral compression fracture with osteopenia: a systematic review and meta-analysis.

BACKGROUND: Surgical and non-surgical interventions are the two categories for treatment of vertebral compression fractures (VCFs). However, there is clinical uncertainty over optimal management. This study aimed to examine the safety and effectiveness of surgical management for treatment of VCFs with osteopenia compared with non-surgical treatment. METHODS: We conducted a systematic search through electronic databases from inception to June 2014, with no limits on study data or language. Randomized controlled trials (RCTs) evaluating surgical versus non-surgical interventions for treatment o

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… Voormolen MH, Mali WP, Lohle PN, Fransen H, Lampmann LE, van der Graaf Y, et al. Percutaneous vertebroplasty compared with optimal pain medication treatment: short-term clinical outcome of patients with subacute or chronic painful osteoporotic vertebral compression fractures. The VERTOS study. …

Current State of the Art and Prospects of T Cell-Redirecting Bispecific Antibodies in Multiple Myeloma.

Multiple myeloma (MM) patients eventually develop multi-drug-resistant disease with poor survival. Hence, the development of novel treatment … th a BsAb and another agent, such as a CD38-targeting antibody or an immunomodulatory drug (e.g., pomalidomide), to further improve response depth and duration. Additionally, the combination of two BsAbs, simultaneously targeting two different antigens to prevent antigen escape, is being explored in clinical studies. The evaluation of BsAbs in earlier …

Utilities measured by rating scale, time trade-off, and standard gamble: review and reference for health care professionals.

Utility is a simple expression of health-related quality of life in individuals with different states of health. A number of studies on utility measurements were conducted and published in the past. We retrieved 164 English-language articles which appeared in 1966 through 1999 for a systematic review. The number of reports has been increasing at an accelerating pace, especially during the past decade. The most widely used method of utility measurement was time trade-off, TTO (40%), followed by rating scale, RS (31%) and standard gamble, SG (29%). The utility of chronic health status was more f

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… Med Decis Making , 1998 ; 18 : 381 - 390 . 10372580 10.1177/0272989X9801800405 13 Bosch JL , Tetteroo E , Mali WP , Hunink MG . Iliac arterial occlusive disease: cost-effectiveness analysis of stent placement versus percutaneous transluminal angioplasty. Dutch Iliac Stent Trial Study Group . …

Nostoc commune-derived scytonemin induced mitochondrial cell death in leukemia models.

Cyanobacteria have long attracted scientific interest through their potential application in the development of new therapeutic approaches, particularly those related to the treatment of cancer. In this study, the antiproliferative effects of Nostoc commune extract (NOS) and the cyanobacterial compound scytonemin (SCY) were evaluated against a variety of in vitro cancer models, including cervix, colon, breast, lung, and leukemia cell lines, using resazurin assays. Both of the studied compounds were found to have inhibited metabolic activity in a dose-dependent manner, with IC50 values ranging

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… Mali BS. Cancer treatment: role of natural products. Time to have a serious rethink. Oral Oncol Rep. 2023;6:100040. 10.1016/j.oor.2023.100040. 3. Chunarkar-Patil P, Kaleem M, Mishra R, Ray S, Ahmad A, Verma D, Bhayye S, Dubey R, Singh HN, Kumar S. …

Circular RNA as a Novel Biomarker for Diagnosis and Prognosis and Potential Therapeutic Targets in Multiple Myeloma.

Circular RNAs (circRNAs) are a novel type of covalently closed RNAs involved in several physiological and pathological processes. They display tissue-specific expression and are constant, abundant, and highly conserved, making them perfect markers for diagnosis and prognosis. Several studies have proposed that circRNAs are also differentially produced in malignancies where they have oncogenic effects. Furthermore, circRNAs affecting microRNAs modify the expression profile of several transcription factors which play essential roles in tumors. CircRNAs within the hematopoietic compartment were i…

Tumor Cell Survival Factors and Angiogenesis in Chronic Lymphocytic Leukemia: How Hot Is the Link?

Chronic lymphocytic leukemia (CLL) is characterized by the accumulation of neoplastic CD5+/CD19+ B lymphocytes in the blood. These cells migrate to and proliferate in the bone marrow and lymphoid tissues. Despite the development of new therapies for CLL, drug resistance and disease relapse still occur; novel treatment approaches are therefore still needed. Inhibition of the angiogenesis involved in the progression of CLL might be a relevant therapeutic strategy. The literature data indicate that vascular endothelial growth factor, angiopoietin-2, and matrix metalloproteinase-9 are pro-angiogen

Management of Acute Myeloid Leukemia: Current Treatment Options and Future Perspectives.

Treatment of acute myeloid leukemia (AML) has improved in recent years and several new therapeutic options have been approved. Most of them include mutation-specific approaches (e.g., gilteritinib for AML patients with activating FLT3 mutations), or are restricted to such defined AML subgroups, such as AML-MRC (AML with myeloid-related changes) or therapy-related AML (CPX-351). With this review, we aim to present a comprehensive overview of current AML therapy according to the evolved spectrum of recently approved treatment strategies. We address several aspects of combined epigenetic therapy

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… Mali R.S., Zhang Q., DeFilippis R., Cavazos A., Kuruvilla V.M., Raman J., Mody V., Choo E.F., Dail M., Shah N.P., et al. Venetoclax combines synergistically with FLT3 inhibition to effectively target leukemic cells in FLT3-ITD+ acute myeloid leukemia models. …

Acute lymphoblastic leukemia: an update on treatment and Brazilian perspective.

Acute lymphoblastic leukemia (ALL) is the most common pediatric cancer, representing a major global health concern, particularly in developing countries. This review provides an updated overview of the epidemiology, diagnosis, prognosis, treatment, and a Brazilian perspective for ALL. Despite advances in chemotherapy and immunotherapy, survival disparities remain challenging, driven by socioeconomic and healthcare access inequalities. Present diagnosis incorporates immunophenotyping, cytogenetics, and molecular biology for precise stratification and treatment planning. Treatment has evolved fr

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… The drugs are very similar to those used in different countries, such as India, Ireland, Tanzania, Thailand and Mali, except for some tyrosine kinase inhibitor and PEG-asparaginase, which are included in other protocols ( 45 ). …

The landscape and trajectory of global CRISPR therapeutics.

With the first regulatory approval in 2023 of a CRISPR-based therapy for sickle cell disease followed by the recent demonstration of an accelerated, personalized CRISPR treatment for congenital severe carbamoyl-phosphate synthetase 1 deficiency, CRISPR-Cas9 genome editing has progressed from a laboratory technology into a clinical reality in just over a decade. While enthusiasm for deploying CRISPR-Cas9 to treat a range of human genetic diseases continues to grow, broad clinical application remains constrained due to technical, regulatory, manufacturing and economic challenges. Here, scientist

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… Multiplex genome engineering using CRISPR/Cas systems Science 339 2013 819 823 10.1126/science.1231143 23287718 PMC3795411 4 Mali P. Yang L. Esvelt K.M. Aach J. Guell M. DiCarlo J.E. Norville J.E. Church G.M. …

Blocking the IL‑6 pathway to treat immune checkpoint inhibitor‑induced inflammatory arthritis (Review).

The incorporation of immune checkpoint inhibitors (ICIs) into cancer treatment has revolutionized oncology, providing marked advantages in managing various types of cancer. Nevertheless, the increasing use of ICIs has led to the emergence of immune‑related side effects, including autoimmune diseases such as inflammatory arthritis. IL‑6 signaling is crucial in the development of inflammatory arthritis and is linked to both the benefits and adverse effects of ICIs. The present review summarizes the latest progress in the IL‑6 pathway in inflammatory arthritis and discusses the therapeutic potent

Проблемы с многократной терапией миеломы у пациентов постарше и посторонних.

Каждый год больше, чем 150,000 во всем мире диагностируются случаи множественной миеломы (ММ) и более 100,000 Зарегистрированы случаи смерти в результате этого злонамеренного поведения. Уровень заболеваемости ММ растет во всем мире, особенно в странах с высоким уровнем дохода и среди мужского населения в возрасте ≥ 50. Хотя прогресс в осуществлении стратегий лечения привел к повышению уровня выживания за последние десятилетия, ММ остается неизлечимым во многих случаях. Важно то, что one- треть пациентов с диагнозом MM - >75 возраст и характеризуется относительно низкой трудоспособностью или сл

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Every year, more than 150,000 cases of multiple myeloma (MM) are diagnosed worldwide, and over 100,000 deaths caused by this malignancy are recorded. MM incidence is increasing globally, particularly in high-income countries and in the male population aged ≥ 50. While advances in treatment strategies have led to the improvement of survival over the past deca…

Interventional Procedures for Vertebral Diseases: Spinal Tumor Ablation, Vertebral Augmentation, and Basivertebral Nerve Ablation-A Scoping Review.

Low back pain is consistently documented as the most expensive and leading cause of disability. The majority of cases have non-specific etiologies. However, a subset of vertebral diseases has well-documented pain generators, including vertebral body tumors, vertebral body fractures, and vertebral endplate injury. Over the past two decades, specific interventional procedures targeting these anatomical pain generators have been widely studied, including spinal tumor ablation, vertebral augmentation, and basivertebral nerve ablation. This scoping review summarizes safety and clinical efficacy and

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… Mali W. Lohle P. Fransen H. Lampmann L. van der Graaf Y. Juttmann J.R. Jansssens X. Verhaar H.J. Percutaneous vertebroplasty compared with optimal pain medication treatment: Short-term clinical outcome of patients with subacute or chronic painful osteoporotic vertebral compression fractures. …

Drug repurposing in malignant pleural mesothelioma: a breath of fresh air?

Drug repurposing is the use of known drugs for new indications. Malignant pleural mesothelioma (MPM) is a rare cancer with a poor prognosis. So far, few treatments have been approved in this disease. However, its incidence is expected to increase significantly, particularly in developing countries. Consequently, drug repurposing appears as an attractive strategy for drug development in MPM, since the known pharmacology and safety profile based on previous approvals of repurposed drugs allows for faster time-to-market for patients and lower treatment cost. This is critical in low- and middle-in…