Article
Hematology
Alexis Leonard, Akshay Sharma, Naoya Uchida, David Stroncek, Sandhya R. Panch, Kamille West, Eoghan Molloy, Thomas E. Hughes, Sara Hauffe, Tiffani Taylor, Courtney Fitzhugh, Jane S. Hankins, Megan Wilson, Shengdar Q. Tsai, Mitchell J. Weiss, Matthew Hsieh, John F. Tisdale
Summary: Recent studies have shown that plerixafor mobilization and apheresis in patients with sickle cell disease can safely collect sufficient CD34(+) hematopoietic stem cells for clinical gene therapy applications. The quantity of CD34(+) cells mobilized by plerixafor varies among SCD patients for unknown reasons, and factors influencing this yield include age, baseline and pre-apheresis CD34(+) cell counts, and disease severity markers unique to SCD.
Article
Oncology
Eric Huselton, Michael P. Rettig, Theresa Fletcher, Julie Ritchey, Leah Gehrs, Kyle McFarland, Stephanie Christ, William C. Eades, Kathryn Trinkaus, Rizwan Romee, Shashikant Kulkarni, Armin Ghobadi, Camille Abboud, Amanda F. Cashen, Keith Stockerl-Goldstein, Geoffrey L. Uy, Ravi Vij, Peter Westervelt, John F. DiPersio, Mark A. Schroeder
Summary: The interaction between the bone marrow microenvironment and MDS tumor clones play an important role in disease pathogenesis and response to treatment. The combination therapy of plerixafor, G-CSF, and azacitidine shows promising response rates in MDS patients, with evidence of mobilization correlating with a higher overall response rate.
LEUKEMIA & LYMPHOMA
(2021)
Article
Immunology
Rebecca A. Sosa, Thomas Mone, Bita V. Naini, Donald B. Kohn, Elaine F. Reed, Kristina Wheeler, Beatriz Campo-Fernandez, Alejandra Davila, Donald J. Chaffin, Joseph DiNorcia, Fady M. Kaldas, Aaron Cohen, Erik L. Lum, Jeffrey L. Veale, Neil M. Kogut
Summary: Solid organ transplantation is the therapy of choice for end-stage organ failure. However, recipients are prone to infections and cancer due to lifelong immunosuppression. The study explores the use of hematopoietic stem cells to induce tolerance in deceased donor kidney recipients, while addressing the concern of immune cell trafficking into the liver.
Article
Cell Biology
Seyede-Leila Shafiei, Aliakbar Movassaghpour, Seyede Fatemeh Hosseini, Mehdi Talebi, Mehdi Edalati, Saeedeh Torabi Goudarzi, Mohammad Sadegh Soltani-Zangbar, Amir Mehdizadeh, Mehdi Yousefi
Summary: This study investigates the factors affecting the homing of hematopoietic stem cells (HSCs) in bone marrow (BM) donors of acute lymphoblastic leukemia (ALL) patients after granulocyte colony-stimulating factor (G-CSF) injection, as well as the effects of G-CSF on BM transplantation quality in these patients. The results show that G-CSF increases the level of CD34+ HSCs and alters the expression of homing factors, leading to improved BM transplantation quality in ALL patients.
CELL BIOLOGY INTERNATIONAL
(2022)
Article
Hematology
Laura Cooling, Justin Kelley, Edison Sexton, Sarah Anand, Sandra Hoffmann
Summary: This article presents a unique case of treatment-related acute lymphoblastic leukemia (t-ALL) that was unmasked by G-CSF during autologous hematopoietic progenitor cell collection. The patient had a history of chemotherapy and developed leukemia as a late complication.
Article
Oncology
Sung-Soo Park, Seung-Hwan Shin, Jung-Yeon Lee, Young-Woo Jeon, Seung-Ah Yhang, Chang-Ki Min
Summary: This study compared the mobilization effects and safety of single-dose etoposide plus G-CSF versus G-CSF alone with risk-adapted plerixafor in MM patients. The findings showed that the etoposide group had a higher count of CD34+ cells collected, but there was no significant difference in the optimal collection rate between the two groups. The etoposide group had higher rates of thrombocytopenia and nausea, but overall adverse events were low in both groups.
Article
Oncology
Vijendra Singh, Hyejeong Jang, Seongho Kim, Lois Ayash, Asif Alavi, Voravit Ratanatharathorn, Joseph P. Uberti, Abhinav Deol
Summary: The use of G-CSF post allogeneic transplant can accelerate neutrophil engraftment and shorten hospital stay, but may also increase the incidence of chronic GVHD and the economic burden of associated complications.
LEUKEMIA & LYMPHOMA
(2021)
Article
Pediatrics
Guadalupe Gonzalez-Villarreal, Myrna Pequeno-Luevano, Severiano Baltazar-Arellano, Adriana Sandoval, Guillermo Sotomayor-Duque, Gerardo Martinez-Pozos, Andres Ortega, Rosa de Leon, Roberto Hernandez
Summary: Haplo-SCT could be considered as a primary treatment option for severe aplastic anemia in children without a matched related or unrelated donor. Using peripheral blood stem cells as the sole source for transplantation has shown to be effective, with post-transplant cyclophosphamide being a good strategy for preventing graft versus host disease.
PEDIATRIC TRANSPLANTATION
(2021)
Article
Hematology
Ahmet Sarici, Mehmet Ali Erkurt, Irfan Kuku, Emin Kaya, Ilhami Berber, Soykan Bicim, Emine Hidayet, Ahmet Kaya, Muhammed Furkan Keser, Omer Faruk Bahcecioglu, Ayss Uysal
Summary: The study aimed to determine the effect of G-CSF on platelet engraftment time after HSCT. The results showed that while G-CSF shortened the duration of neutrophil engraftment and hospitalization, it increased platelet engraftment time. The incidence of acute GVHD tended to be higher in the group receiving G-CSF. Further investigation is needed to understand the effects of G-CSF on platelet engraftment.
TRANSFUSION AND APHERESIS SCIENCE
(2022)
Review
Biochemistry & Molecular Biology
Jennifer E. Adair, Lindsay Androski, Lois Bayigga, Deus Bazira, Eugene Brandon, Lynda Dee, Steven Deeks, Mohammed Draz, Karine Dube, Mark Dybul, Umut Gurkan, Evelyn Harlow, Cissy Kityo, Michael Louella, Punam Malik, Vikram Mathews, Adrian McKemey, Henry Mugerwa, Daniel Muyanja, Olabimpe Olayiwola, Rimas J. Orentas, Alex Popovski, Jeff Sheehy, Francis Ssali, Moses Supercharger Nsubuga, John F. Tisdale, Els Verhoeyen, Boro Dropulic
Summary: The gene and cell therapy field has seen significant growth, but there is a geographical exclusion of low- and middle-income countries. To address this, the Global Gene Therapy Initiative aims to introduce clinical trials in Uganda and India.
Article
Genetics & Heredity
Petros Kountouris, Coralea Stephanou, Carsten W. Lederer, Joanne Traeger-Synodinos, Celeste Bento, Cornelis L. Harteveld, Eirini Fylaktou, Tamara T. Koopmann, Hashim Halim-Fikri, Kyriaki Michailidou, Landry E. Nfonsam, John S. Waye, Bin A. Zilfalil, Marina Kleanthous
Summary: This article discusses the experience of the ClinGen Variant Curation Expert Panel in adapting ACMG/AMP criteria for classifying variants in three globin genes related to recessively inherited hemoglobinopathies, using five evidence categories as examples to demonstrate the process of specification and rationale underlying the classification.
Article
Biophysics
Eleni Gavriilaki, Panagiota Anyfanti, Ioanna Sakellari, Ioannis Batsis, Panagiotis Dolgyras, Antonios Lazaridis, Barbara Nikolaidou, Nikolaos Koletsos, Maria Gavriilaki, Ippokratis Zarifis, Marianna Masmanidou, Zoi Bousiou, Anna Vardi, Stella Douma, Achilles Anagnostopoulos, Eugenia Gkaliagkousi
Summary: This study found impaired microvascular function in allogeneic hematopoietic cell transplantation (alloHCT) survivors free of graft-versus-host-disease or relapse, independently of cardiovascular risk factors. The study suggests the need for further research on novel markers for cardiovascular risk prediction and the effects of disease type, phase, and pre-transplant treatments.
BONE MARROW TRANSPLANTATION
(2022)
Article
Oncology
Chrysavgi Lalayanni, Eleni Gavriilaki, Anastasia Athanasiadou, Michael Iskas, Maria Papathanasiou, Anastasia Marvaki, Sotiria Mpesikli, Giorgos Papaioannou, Despina Mallouri, Ioannis Batsis, Apostolia Papalexandri, Ioanna Sakellari, Achilles Anagnostopoulos
Summary: Therapy related acute myeloid leukemia (tAML) and secondary AML after an antecedent hematologic disorder (sAML-AHD) are often considered together, although they have some clinical and prognostic differences. Both sAML groups have poor outcomes after uniform and intensive treatment. Allogeneic hematopoietic cell transplantation (alloHCT) was found to be an independent predictor of outcome in both groups, while karyotype only affected sAML-AHD.
CLINICAL LYMPHOMA MYELOMA & LEUKEMIA
(2022)
Correction
Biochemistry & Molecular Biology
Jennifer E. Adair, Lindsay Androski, Lois Bayigga, Deus Bazira, Eugene Brandon, Lynda Dee, Steven Deeks, Mohamed Draz, Karine Dube, Mark Dybul, Umut Gurkan, Evelyn Harlow, Cissy Kityo, Michael Louella, Punam Malik, Vikram Mathews, Adrian McKemey, Henry Mugerwa, Daniel Muyanja, Olabimpe Olayiwola, Rimas J. Orentas, Alex Popovski, Jeff Sheehy, Francis Ssali, Moses Supercharger Nsubuga, John F. Tisdale, Els Verhoeyen, Boro Dropulic
Article
Biochemistry & Molecular Biology
Panayiota L. Papasavva, Petros Patsali, Constantinos C. Loucari, Ryo Kurita, Yukio Nakamura, Marina Kleanthous, Carsten W. Lederer
Summary: Spatial and temporal precision of genetic intervention greatly benefits molecular therapies and functional studies. This study explores tag-activated microRNA (miRNA)-mediated endogene deactivation for research and potential lineage-specific therapy. The results demonstrate the suitability of this method for physiologically relevant modulation of gene expression, but its current form is not suitable for therapeutic applications.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Cell Biology
Panagiotis G. Asteris, Eleni Gavriilaki, Tasoula Touloumenidou, Evaggelia-Evdoxia Koravou, Maria Koutra, Penelope Georgia Papayanni, Alexandros Pouleres, Vassiliki Karali, Minas E. Lemonis, Anna Mamou, Athanasia D. Skentou, Apostolia Papalexandri, Christos Varelas, Fani Chatzopoulou, Maria Chatzidimitriou, Dimitrios Chatzidimitriou, Anastasia Veleni, Evdoxia Rapti, Ioannis Kioumis, Evaggelos Kaimakamis, Milly Bitzani, Dimitrios Boumpas, Argyris Tsantes, Damianos Sotiropoulos, Anastasia Papadopoulou, Ioannis G. Kalantzis, Lydia A. Vallianatou, Danial J. Armaghani, Liborio Cavaleri, Amir H. Gandomi, Mohsen Hajihassani, Mahdi Hasanipanah, Mohammadreza Koopialipoor, Paulo B. Lourenco, Pijush Samui, Jian Zhou, Ioanna Sakellari, Serena Valsami, Marianna Politou, Styliani Kokoris, Achilles Anagnostopoulos
Summary: This study aimed to develop a predictive model for ICU hospitalization and death in COVID-19 patients and validate the association between complement-related genetic variants and impaired complement phenotype. We identified critical variants associated with severe COVID-19 and developed an artificial neural network (ANN) predicting morbidity and mortality with high accuracy.
JOURNAL OF CELLULAR AND MOLECULAR MEDICINE
(2022)
Article
Medicine, General & Internal
Christos Varelas, Eleni Gavriilaki, Ioanna Sakellari, Philippos Klonizakis, Evaggelia-Evdoxia Koravou, Ioanna Christodoulou, Ioulia Mavrikou, Andreas Kourelis, Fani Chatzopoulou, Dimitrios Chatzidimitriou, Tasoula Touloumenidou, Apostolia Papalexandri, Achilles Anagnostopoulos, Efthimia Vlachaki
Summary: Most SCD patients demonstrated adequate levels of neutralizing antibodies against SARS-CoV-2 after the second dose of mRNA vaccines, with a certain association with complement activation. Further studies are critical to determine the durability of this immune response and the potential benefit of a third dose.
JOURNAL OF CLINICAL MEDICINE
(2022)
Review
Pharmacology & Pharmacy
Carsten W. Lederer, Lola Koniali, Tina Buerki-Thurnherr, Panayiota L. Papasavva, Stefania La Grutta, Amelia Licari, Frantisek Staud, Donato Bonifazi, Marina Kleanthous
Summary: Advanced therapy medicinal products show great promise in pediatric applications, with potential to improve treatment outcomes for untreatable disorders and inherited diseases. Early application in children may provide better results and reduce overall requirements.
Article
Hematology
Julia H. Joo, Xuefeng Wang, Swati Singh, Chun -Yu Chen, Chong Li, Jennifer E. Adair, Hans -Peter Kiem, David J. Rawlings, Carol H. Miao
Summary: This study demonstrates the long-term safety and efficacy of intraosseous delivery of lentiviral vector for factor VIII in treating hemophilia A in a humanized model. The successful transduction of self-regenerating hematopoietic stem and progenitor cells was confirmed, indicating potential for long-term transgene expression. The study also evaluated the safety and efficacy of gene transfer therapy into human HSPCs, showing megakaryocyte-specific gene expression and polyclonal integration pattern. Overall, the results support the feasibility of translating this method for clinical applications.
Letter
Biophysics
Raffaella Greco, Jorinde D. Hoogenboom, Edouard F. Bonneville, Achilles Anagnostopoulos, Angela Cuoghi, Jean-Hugues Dalle, Eva M. Weissinger, Peter Lang, Federica Galaverna, Massimo Martino, Alexei Maschan, Christine Mauz-Korholz, Maddalena Noviello, Jakob Passweg, Jacopo Peccatori, Montserrat Rovira, Carlos Solano, Hendrik Veelken, Andrea Velardi, Eva Maria Wagner-Drouet, Xi Zhang, Fabio Ciceri, Chiara Bonini, Luca Vago, Annalisa Ruggeri, Christian Chabannon
BONE MARROW TRANSPLANTATION
(2023)
Review
Biochemistry & Molecular Biology
Kiriaki Paschoudi, Evangelia Yannaki, Nikoletta Psatha
Summary: Beta-hemoglobinopathies, the most common genetic disorders globally, can be cured by gene therapy or genome editing approaches. Gene therapy involves introducing therapeutic genes into patients' stem cells, while genome editing targets specific mutations for disease modification. These approaches have shown promising outcomes in increasing transfusion independence and reducing symptoms. Ongoing research on new HbF modulators and long-term follow-up studies are needed for further confirmation.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Oncology
Maria Alvanou, Memnon Lysandrou, Panayota Christophi, Nikoleta Psatha, Alexandros Spyridonidis, Anastasia Papadopoulou, Evangelia Yannaki
Summary: T-cell-based, personalized immunotherapy has become the mainstream treatment for certain blood cancers and shows potential for further indications. However, CAR-T cell therapies, which are genetically modified T-cell therapies targeting specific antigens, face challenges in providing durable responses and achieving success in treating solid cancers. These challenges include poor persistence, impaired tumor trafficking, and difficulties in penetrating the hostile tumor microenvironment. Increasing evidence suggests that epigenetic regulation plays a role in the in vivo performance of CAR-T cells. Understanding the impact of epigenetic reprogramming on T-cell differentiation, exhaustion, and tumor infiltration may lead to the development of more potent CAR-T immunotherapies.
Article
Oncology
Eleni Gavriilaki, Panagiotis Dolgyras, Sotiria Dimou-Mpesikli, Aikaterini Poulopoulou, Paschalis Evangelidis, Nikolaos Evangelidis, Christos Demosthenous, Evangelia Zachrou, Panagiotis Siasios, Despina Mallouri, Anna Vardi, Zoi Bousiou, Alkistis Panteliadou, Ioannis Batsis, Marianna Masmanidou, Chrysavgi Lalayanni, Evangelia Yannaki, Damianos Sotiropoulos, Achilles Anagnostopoulos, Timoleon-Achilleas Vyzantiadis, Ioanna Sakellari
Summary: This retrospective study investigated the prevalence and outcome of invasive fungal infections (IFD) in patients who received cellular therapies. It was found that CAR T cell and gene therapy recipients did not develop IFD, while autologous HCT recipients with primary refractory/relapsed lymphomas had probable IFD. Among allogeneic HCT recipients, 11 experienced probable IFD, 31 had possible IFD, and 10 had proven IFD. These findings suggest that IFD is associated with poor outcomes in allogeneic HCT recipients.
Article
Biotechnology & Applied Microbiology
Nikoletta Y. Papaioannou, Petros Patsali, Basma Naiisseh, Panayiota L. Papasavva, Lola Koniali, Ryo Kurita, Yukio Nakamura, Soteroula Christou, Maria Sitarou, Claudio Mussolino, Toni Cathomen, Marina Kleanthous, Carsten W. Lederer
Summary: Genome editing tools have been successfully used for gene therapy and reverse genetics. However, the challenges lie in the efficient delivery of editors to target cells and sites and the independence from commercially available tools. This study presents an in vitro transcription protocol for RNA-based delivery of genome editing tools, which allows high editing efficiencies and flexibility in application to different editors.
FRONTIERS IN GENOME EDITING
(2023)
