News Item
Biotechnology & Applied Microbiology
Cormac Sheridan
Summary: Early results show promise for a one-off treatment for sickle cell disease, but gene-editing and gene therapies must demonstrate long-lasting protection and affordability for a global patient population.
NATURE BIOTECHNOLOGY
(2021)
Review
Medicine, General & Internal
Yvette C. Tanhehco, Ghazala Nathu, Ljiljana V. Vasovic
Summary: Recent advances in managing Sickle Cell Disease (SCD) have significantly improved patient survival and quality of life. Disease-modifying drug therapies, allogeneic hematopoietic stem cell transplantation, and gene therapy have shown efficacy in reducing pain crises and severe complications. Multidisciplinary expertise is crucial in developing the best treatment strategy for SCD patients.
FRONTIERS IN MEDICINE
(2022)
Review
Medicine, General & Internal
Fahd A. Kuriri
Summary: This article provides an overview of current, new, and future treatment options for sickle cell disease (SCD). Current treatments include hydroxyurea and voxelotor, which increase fetal hemoglobin levels and selectively bind hemoglobin, respectively. Potential side effects and the need for new treatments are discussed, along with the promise of gene therapy as a future treatment option for SCD.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Multidisciplinary Sciences
Xin Gao, Dachuan Zhang, Chunliang Xu, Huihui Li, Kathleen M. Caron, Paul S. Frenette
Summary: Nociceptive nerves play a role in enforcing HSC mobilization through CGRP secretion, collaborating with sympathetic nerves to maintain HSCs in the bone marrow. CGRP directly activates HSCs via the G alpha(s)/adenylyl cyclase/cAMP pathway, and consumption of capsaicin-containing food enhances HSC mobilization in mice.
Article
Multidisciplinary Sciences
Gregory A. Newby, Jonathan S. Yen, Kaitly J. Woodard, Thiyagaraj Mayuranathan, Cicera R. Lazzarotto, Yichao Li, Heather Sheppard-Tillman, Shaina N. Porter, Yu Yao, Kalin Mayberry, Kelcee A. Everette, Yoonjeong Jang, Christopher J. Podracky, Elizabeth Thaman, Christophe Lechauve, Akshay Sharma, Jordana M. Henderson, Michelle F. Richter, Kevin T. Zhao, Shannon M. Miller, Tina Wang, Luke W. Koblan, Anton P. McCaffrey, John F. Tisdale, Theodosia A. Kalfa, Shondra M. Pruett-Miller, Shengdar Q. Tsai, Mitchell J. Weiss, David R. Liu
Summary: The study demonstrated successful conversion of sickle cell disease allele into a non-pathogenic variant using adenine base editor, with durable therapeutic effects. The edited HSPCs improved physiological parameters and reduced pathological abnormalities in spleens of mice, indicating the potential for long-lasting and effective treatment for SCD.
Review
Genetics & Heredity
Merlin Crossley, Georgios E. Christakopoulos, Mitchell J. Weiss
Summary: Sickle cell disease is a common genetic blood disorder that causes acute and chronic pain, multiorgan damage, and early death. Recent advancements in technology and research have made curing most patients possible, and there is ongoing exploration of more accessible treatments.
TRENDS IN GENETICS
(2022)
Article
Immunology
Luciana Ribeiro Jarduli-Maciel, Julia Teixeira Cottas de Azevedo, Emmanuel Clave, Thalita Cristina de Mello Costa, Lucas Coelho Marliere Arruda, Isabelle Fournier, Patricia Vianna Bonini Palma, Keli Cristina Lima, Juliana Bernardes Elias, Ana Beatriz P. L. Stracieri, Fabiano Pieroni, Renato Cunha, Luiz Guilherme Darrigo-Junior, Carlos Eduardo Settani Grecco, Dimas Tadeu Covas, Ana Cristina Silva-Pinto, Gil Cunha De Santis, Belinda Pinto Simoes, Maria Carolina Oliveira, Antoine Toubert, Kelen Cristina Ribeiro Malmegrim
Summary: In this study, the reconstitution of T- and B-cell compartments in SCD patients treated with allo-HSCT was comprehensively evaluated. The occurrence of acute graft-versus-host disease transiently affected the reconstitution of T- and B-cells. In addition, an increase in IL-10-producing B-regulatory cells was observed after transplantation, which may contribute to improved immune regulation and homeostasis.
CLINICAL & TRANSLATIONAL IMMUNOLOGY
(2022)
Review
Biochemistry & Molecular Biology
Seahyoung Lee, Dong-Sik Chae, Byeong-Wook Song, Soyeon Lim, Sang Woo Kim, Il-Kwon Kim, Ki-Chul Hwang
Summary: Recent clinical trials have shown that one-third of studies on adipose-derived stem cells (ADSCs) focused on musculoskeletal disorders (MSD). These disorders are a major burden to society and conventional treatments are often inadequate, leading to the investigation of ADSC-based cell therapies as more effective alternatives. Categorizing the specific MSDs targeted by ADSC therapy and understanding their mechanisms of action can aid in developing optimized strategies for ADSC-based treatments in the future.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Review
Pediatrics
Katharina Kleinschmidt, Meng Lv, Asaf Yanir, Julia Palma, Peter Lang, Matthias Eyrich
Summary: Allogeneic hematopoietic stem cell transplantation (HSCT) is a potential curative option for high-risk or refractory/relapsed leukemias in children, with haploidentical HSCT (hHSCT) showing significant advantages but posing the key question of how to deplete donor T-cells effectively. In vivo T-cell suppression and ex vivo T-cell depletion are two established methods to reduce alloreactivity in hHSCT, with different protocols utilizing post-transplantation cyclophosphamide or anti-thymocyte globulin. Studies have not shown a clear benefit for any specific method in terms of overall survival, non-relapse mortality, or disease recurrence.
FRONTIERS IN PEDIATRICS
(2021)
Review
Biochemistry & Molecular Biology
Luca Marsili, Jennifer Sharma, Tiago Fleming Outeiro, Carlo Colosimo
Summary: Stem cell-based therapies (SCT) have potential in treating neurodegenerative disorders, but clinical trials are just starting and results may take several years. SCTs can provide both symptomatic and disease-modifying effects, and may complement molecular therapies in precision medicine.
Article
Clinical Neurology
Alice Mariottini, Giovanni Bulgarini, Benedetta Forci, Chiara Innocenti, Fabrizia Mealli, Alessandra Mattei, Chiara Ceccarelli, Anna Maria Repice, Alessandro Barilaro, Claudia Mechi, Riccardo Saccardi, Luca Massacesi
Summary: The study demonstrates that for patients with secondary-progressive multiple sclerosis (SP-MS), AHSCT is more effective than cyclophosphamide (Cy) in reducing relapse activity, but there is no significant difference between the two in terms of disability progression. AHSCT is beneficial for reducing relapses, but disability progression in SP-MS is more likely driven by noninflammatory neurodegeneration.
EUROPEAN JOURNAL OF NEUROLOGY
(2022)
Article
Hematology
Stefan Cordes, Chuanfeng Wu, Cynthia E. Dunbar
Summary: Recent advances in high-throughput genomics have allowed for direct tracking of outputs from various cell types, greatly speeding up research on developmental processes and tissue regeneration. Understanding the biology and clonal dynamics of cells in haematopoiesis is crucial for comprehending the response to aging, malignant transformation, and for designing more effective and durable clinical gene and cellular therapies.
BRITISH JOURNAL OF HAEMATOLOGY
(2021)
Review
Rheumatology
Dinesh Khanna, Nancy Krieger, Keith M. Sullivan
Summary: This narrative review examines the evolving cell therapies for the treatment of diffuse cutaneous scleroderma (dcSSc) and other autoimmune diseases, including mesenchymal stem cells, chimeric antigen receptor-based therapy, tolerogenic dendritic cells, and facilitating cells.
Review
Cell & Tissue Engineering
Ahmed Lotfy, Aya Elgamal, Anna Burdzinska, Ayman A. Swelum, Reham Soliman, Ayman A. Hassan, Gamal Shiha
Summary: Autoimmune hepatitis is a chronic inflammatory liver disease, and adult stem cells (ASCs) have been investigated as potential therapies with positive effects. Hematopoietic stem cells (HSCs) and mesenchymal stem cells (MSCs) interact with immune response pathways and are potentially valuable in treating autoimmune diseases like autoimmune hepatitis.
STEM CELL RESEARCH & THERAPY
(2021)
Article
Oncology
Mary Eapen, Ruta Brazauskas, David A. Williams, Mark C. Walters, Andrew St Martin, Benjamin L. Jacobs, Joseph H. Antin, Kira Bona, Sonali Chaudhury, Victoria H. Coleman-Cowger, Nancy L. DiFronzo, Erica B. Esrick, Joshua J. Field, Courtney D. Fitzhugh, Julie Kanter, Neena Kapoor, Donald B. Kohn, Lakshmanan Krishnamurti, Wendy B. London, Michael A. Pulsipher, Sohel Talib, Alexis A. Thompson, Edmund K. Waller, Ted Wun, Mary M. Horowitz
Summary: This study investigates the incidence and risk factors for secondary neoplasm after transplantation for sickle cell disease. The results show that the 10-year incidence of leukemia/MDS was 1.7% and of any secondary neoplasm was 2.4%. Low-intensity regimens were associated with higher risks for leukemia/MDS or any secondary neoplasm compared with more intense regimens.
JOURNAL OF CLINICAL ONCOLOGY
(2023)
