Review
Genetics & Heredity
Laura FitzPatrick, Adrian Bird
Summary: The review highlights the genetic basis of many neurological disorders and the efforts being made to develop genetic strategies for treating monogenic neurological disorders. It also addresses the challenges and ethical difficulties that may arise in the process.
Review
Virology
Valentina Poletti, Fulvio Mavilio
Summary: Lentiviral vectors are commonly used in gene therapy for monogenic diseases, where they transduce autologous somatic cells in an ex vivo setting for re-delivery to patients. Key applications include hematopoietic stem cells for hematological or neurometabolic diseases and T cells for cancer immunotherapy.
Review
Cell Biology
Ana Sofia Coroadinha
Summary: Gene therapy relies on the delivery of genetic material to the patient's cells to provide therapeutic treatment. Lentiviral (LV) and adeno-associated virus (AAV) vectors are two commonly used and efficient delivery systems. However, the therapeutic efficacy of these vectors is hindered by host restriction factors (RFs) that block their transduction. In this review, we discuss the identified RFs that impede LV and AAV vector transduction and limit their therapeutic effectiveness.
Article
Multidisciplinary Sciences
Piamsiri Sawaisorn, Korakot Atjanasuppat, Kitipong Uaesoontrachoon, Parin Rattananon, Worapapar Treesuppharat, Suradej Hongeng, Usanarat Anurathapan
Summary: T cells genetically engineered to express a chimeric antigen receptor (CAR) specifically binding to a CD19 antigen have shown remarkable antitumor effect in treating B-cell malignancies. This study compares the transduction ability of second and third generations lentiviral vectors (LVV) to improve the efficacy of CD19 CARs. The results demonstrate that third generation LVV transduced T cells have higher transduction efficacy and show increased cytotoxic capacity against target cells. In a mouse model, third generation LVV transduced T cells successfully limit tumor burden and increase the length of survival. Evaluating the efficacy of CAR-CD19 T cells, this study suggests that third generation LVV transduction generates functional CAR-CD19 T cells, offering a safer and effective therapy for B-cell malignancies.
Review
Pharmacology & Pharmacy
Fatima Aerts-Kaya, Niek P. van Til
Summary: Leukodystrophies are a group of genetic degenerative brain disorders that can be lethal if not treated. Gene therapy offers promising results for the treatment of leukodystrophies, and this article discusses the potential interventions of gene therapy in certain types of leukodystrophies, such as X-linked adrenoleukodystrophy and metachromatic leukodystrophy. The advantages and disadvantages of different gene therapy approaches are also discussed, along with the ongoing developments in the field of molecular technologies for treating leukodystrophies in the future.
Article
Immunology
Min-Wen Ku, Pierre Charneau, Laleh Majlessi
Summary: Lentiviral vectors are powerful tools for vaccination, with high efficiency in transducing dendritic cells and inducing long-lasting humoral immunity. They are non-replicative, minimally inflammatory, and novel targets for vaccine development, showing potential in prophylaxis against infectious diseases and onco-immunotherapy.
EXPERT REVIEW OF VACCINES
(2021)
News Item
Multidisciplinary Sciences
Diana Kwon
Summary: Drugs that silence faulty genes could potentially treat brain diseases, but a halted clinical trial has posed a setback in this field.
Article
Immunology
Hang Xie, Ruoxuan Xiang, Hamilton J. Wan, Ewan P. Plant, Peter Radvak, Martina Kosikova, Xing Li, Olga Zoueva, Zhiping Ye, Xiu-Feng Wan
Summary: The study found that postvaccination antibodies induced by the B/Vic vaccine component of the 2019/20 influenza season had reduced HAI cross-reactivity toward predominant 3DEL viruses in the United States. Specifically, antibodies against the 136E+150K subgroup showed significantly lower titers compared to those against the 136E+150N viruses. Close monitoring of the 3DEL 136E+150K subgroup is recommended should it return and predominate the 2020/21 influenza season.
CLINICAL INFECTIOUS DISEASES
(2021)
Review
Clinical Neurology
Iman Khan, Taimoor Asif Jaura, Alaa Tukruna, Aabiya Arif, Sameer Saleem Tebha, Sameen Nasir, Dattatreya Mukherjee, Nadia Masroor, Abubakr Yosufi
Summary: This systematic review evaluated the efficacy and tolerability of alternative drugs for obsessive-compulsive disorder (OCD), and found that Psilocybin, Cannabis, Nicotine, and Morphine may have positive effects in alleviating OCD symptoms. Further research is needed to understand the mechanism of action of these drugs.
NEUROPSYCHIATRIC DISEASE AND TREATMENT
(2023)
Review
Endocrinology & Metabolism
S. Ellison, H. Parker, B. Bigger
Summary: Lysosomal Storage Disorders (LSDs) are inherited diseases caused by defects in specific lysosomal proteins, leading to toxic accumulation of waste products. Most LSDs have no cure and present with progressive neurodegeneration. Overcoming the blood-brain barrier is crucial for effective treatment, and various therapies including gene therapy are being evaluated in clinical trials. If safety and efficacy are demonstrated, these therapies have the potential to become the new standard of care for LSD patients.
JOURNAL OF INHERITED METABOLIC DISEASE
(2023)
Article
Chemistry, Multidisciplinary
Smrithi Padmakumar, Anisha D'Souza, Neha N. Parayath, Benjamin S. Bleier, Mansoor M. Amiji
Summary: Nucleic acid therapeutics, delivered through non-viral vectors, have shown advanced efficacy in treating central nervous system diseases. However, challenges such as the blood-brain barrier, complex pathophysiological changes, and the presence of non-dividing cells need to be overcome for successful therapy. Extensive scientific efforts are being made to optimize non-invasive and safer administration approaches for clinical translation.
JOURNAL OF CONTROLLED RELEASE
(2022)
Article
Multidisciplinary Sciences
Abhirup Bagchi, Nivedhitha Devaraju, Karthik Chambayil, Vignesh Rajendiran, Vigneshwaran Venkatesan, Nilofer Sayed, Aswin Anand Pai, Aneesha Nath, Ernest David, Yukio Nakamura, Poonkuzhali Balasubramanian, Alok Srivastava, Saravanabhavan Thangavel, Kumarasamypet M. Mohankumar, Shaji R. Velayudhan
Summary: This study presents novel lineage-specific lentiviral RNAi vectors that efficiently reduce target gene expression in erythroid cells, making them suitable for gene therapy of hemoglobinopathies and high-throughput RNAi screening for studying normal and pathological erythropoiesis.
SCIENTIFIC REPORTS
(2022)
Review
Medicine, Research & Experimental
Phillip A. Doerfler, Akshay Sharma, Jerlym S. Porter, Yan Zheng, John F. Tisdale, Mitchell J. Weiss
Summary: Sickle cell disease is a monogenic disorder with severe symptoms, but emerging technologies and interdisciplinary collaborations offer potential for more effective treatments. Success relies on ethical considerations, genetic modification of patient HSCs, and close collaboration between researchers, caregivers, and patients.
JOURNAL OF CLINICAL INVESTIGATION
(2021)
Article
Multidisciplinary Sciences
Emilio Gomez-Gonzalez, Beatriz Fernandez-Munoz, Alejandro Barriga-Rivera, Jose Manuel Navas-Garcia, Isabel Fernandez-Lizaranzu, Francisco Javier Munoz-Gonzalez, Ruben Parrilla-Giraldez, Desiree Requena-Lancharro, Manuel Guerrero-Claro, Pedro Gil-Gamboa, Cristina Rosell-Valle, Carmen Gomez-Gonzalez, Maria Jose Mayorga-Buiza, Maria Martin-Lopez, Olga Munoz, Juan Carlos Gomez Martin, Maria Isabel Relimpio Lopez, Jesus Aceituno-Castro, Manuel A. Perales-Esteve, Antonio Puppo-Moreno, Francisco Jose Garcia Cozar, Lucia Olvera-Collantes, Silvia de los Santos-trigo, Emilia Gomez, Rosario Sanchez Pernaute, Javier Padillo-Ruiz, Javier Marquez-Rivas
Summary: Optical spectroscopic techniques are commonly used in the agro-food industry to detect biofilm-forming pathogens, and recently, it has been discovered that near-infrared spectroscopy can also detect viruses in animal and vegetal tissues. A platform based on visible and near-infrared hyperspectral imaging has been developed for non-contact, reagent-free detection and quantification of laboratory-engineered viral particles in fluid samples, with successful detection achieved in phosphate buffered solution and artificial saliva. This innovative approach could potentially be used for rapid mass screening of viral infectious diseases and monitoring of the SARS-CoV-2 pandemic.
SCIENTIFIC REPORTS
(2021)
Review
Medicine, General & Internal
Priyalakshmi Panikker, Shomereeta Roy, Anuprita Ghosh, B. Poornachandra, Arkasubhra Ghosh
Summary: Advancements in genomic medicine have revolutionized the diagnosis and treatment of ocular diseases, particularly retinal diseases. Gene therapy has shown great potential in clinical trials for these diseases, with a focus on personalized diagnostics and therapeutics. This review provides a comprehensive overview of the current state of precision medicine in ophthalmology.
FRONTIERS IN MEDICINE
(2022)
Article
Clinical Neurology
Ide Smets, Barnaby Fiddes, Josselyn E. Garcia-Perez, Di He, Klara Mallants, Wenjia Liao, James Dooley, George Wang, Stephanie Humblet-Baron, Benedicte Dubois, Alastair Compston, Joanne Jones, Alasdair Coles, Adrian Liston, Maria Ban, An Goris, Stephen Sawcer
Article
Allergy
Erika Van Nieuwenhove, Josselyn E. Garcia-Perez, Christine Helsen, Princess D. Rodriguez, Pauline A. van Schouwenburg, James Dooley, Susan Schlenner, Mirjam van der Burg, Els Verhoeyen, Rik Gijsbers, Seth Frietze, Hilde Schjerven, Isabelle Meyts, Frank Claessens, Stephanie Humblet-Baron, Carine Wouters, Adrian Liston
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2018)
Article
Allergy
Jessica Heremans, Josselyn E. Garcia-Perez, Ernest Turro, Susan M. Schlenner, Ingele Casteels, Roxanne Collin, Francis de Zegher, Daniel Greene, Stephanie Humblet-Baron, Sylvie Lesage, Patrick Matthys, Christopher J. Penkett, Karen Put, Kathleen Stirrups, Chantal Thys, Chris Van Geet, Erika Van Nieuwenhove, Carine Wouters, Isabelle Meyts, Kathleen Freson, Adrian Liston
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2018)
Article
Immunology
Josselyn E. Garcia-Perez, Lotte Mathe, Stephanie Humblet-Baron, Annabel Braem, Katrien Lagrou, Patrick Van Dijck, Adrian Liston
FRONTIERS IN IMMUNOLOGY
(2018)
Article
Medicine, Research & Experimental
Biswajit Paul, Guillermo S. Romano Ibarra, Nicholas Hubbard, Teresa Einhaus, Alexander Astrakhan, David J. Rawlings, Hans-Peter Kiem, Christopher W. Peterson
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2018)
Article
Pediatrics
Erika Van Nieuwenhove, Stephanie Humblet-Baron, Lien Van Eyck, Lien De Somer, James Dooley, Thomas Tousseyn, Michael Hershfield, Adrian Liston, Carine Wouters
Article
Rheumatology
Susan Schlenner, Emanuela Pasciuto, Vasiliki Lagou, Oliver Burton, Teresa Prezzemolo, Steffie Junius, Carlos P. Roca, Cyril Seillet, Cynthia Louis, James Dooley, Kylie Luong, Erika Van Nieuwenhove, Ian P. Wicks, Gabrielle Belz, Stephanie Humblet-Baron, Carine Wouters, Adrian Liston
ANNALS OF THE RHEUMATIC DISEASES
(2019)
Article
Hematology
Stephanie Humblet-Baron, John S. Barber, Carlos P. Roca, Aurelie Lenaerts, Pandelakis A. Koni, Adrian Liston
Article
Allergy
Stephanie Humblet-Baron, Dean Franckaert, James Dooley, Fatima Ailal, Aziz Bousfiha, Caroline Deswarte, Carmen Oleaga-Quintas, Jean-Laurent Casanova, Jacinta Bustamante, Adrian Liston
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2019)
Article
Cell Biology
Vasiliki Lagou, Josselyn E. Garcia-Perez, Ide Smets, Lies Van Horebeek, Marijne Vandebergh, Liye Chen, Klara Mallants, Teresa Prezzemolo, Kelly Hilven, Stephanie Humblet-Baron, Matthieu Moisse, Philip Van Damme, Guy Boeckxstaens, Paul Bowness, Benedicte Dubois, James Dooley, Adrian Liston, An Goris
Article
Rheumatology
Erika Van Nieuwenhove, Vasiliki Lagou, Lien Van Eyck, James Dooley, Ulrich Bodenhofer, Carlos Roca, Marijne Vandebergh, An Goris, Stephanie Humblet-Baron, Carine Wouters, Adrian Liston
ANNALS OF THE RHEUMATIC DISEASES
(2019)
Editorial Material
Cell Biology
Adrian Liston, Stephanie Humblet-Baron
IMMUNOLOGY AND CELL BIOLOGY
(2019)
Article
Allergy
Erika Van Nieuwenhove, John S. Barber, Julika Neumann, Elien Smeets, Mathijs Willemsen, Emanuela Pasciuto, Teresa Prezzemolo, Vasiliki Lagou, Laura Seldeslachts, Bert Malengier-Devlies, Mieke Metzemaekers, Sarah Hassdenteufel, Axelle Kerstens, Rob van der Kant, Frederic Rousseau, Joost Schymkowitz, Daniele Di Marino, Sven Lang, Richard Zimmermann, Susan Schlenner, Sebastian Munck, Paul Proost, Patrick Matthys, Christine Devalck, Nancy Boeckx, Frank Claessens, Carine Wouters, Stephanie Humblet-Baron, Isabelle Meyts, Adrian Liston
JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY
(2020)
Article
Immunology
Els Wauters, Karin Thevissen, Carine Wouters, Francesca Maria Bosisio, Frederik De Smet, Jan Gunst, Stephanie Humblet-Baron, Diether Lambrechts, Adrian Liston, Patrick Matthys, Johan Neyts, Paul Proost, Birgit Weynand, Joost Wauters, Sabine Tejpar, Abhishek D. Garg
FRONTIERS IN IMMUNOLOGY
(2020)
Article
Medicine, Research & Experimental
Wai-Hang Leung, Joel Gay, Unja Martin, Tracy E. Garrett, Holly M. Horton, Michael T. Certo, Bruce R. Blazar, Richard A. Morgan, Philip D. Gregory, Jordan Jarjour, Alexander Astrakhan