Editorial Material
Biochemistry & Molecular Biology
Elia R. Lopez, William F. Borschel, Bryan J. Traynor
Summary: Two studies demonstrate the progress of antisense oligonucleotide therapy for amyotrophic lateral sclerosis, offering hope for a viable treatment.
Review
Chemistry, Medicinal
Nhan H. Nguyena, Nicole L. Jarvia, Sathy Balu-Iyera
Summary: The introduction and development of biologics have revolutionized disease treatment, but the immunogenicity issue limits the efficacy for some patients. This review discusses the immunogenicity issue in Hemophilia A therapy as an example, and explores various therapeutic modalities that are being used. Strategies to manage and mitigate immunogenicity are also reviewed.
JOURNAL OF PHARMACEUTICAL SCIENCES
(2023)
Article
Geriatrics & Gerontology
Rosaly Correa-de Araujo, William J. Evans, Roger A. Fielding, Venkatesh Krishnan, Robert H. Carter, James Appleby, Jack Guralnik, Lloyd B. Klickstein, Peter Marks, Alison A. Moore, Sue Peschin, Shalender Bhasin
Summary: Functional limitations and physical disabilities caused by aging and chronic diseases are significant issues that require the expedited development of function-promoting therapies. Collaboration among stakeholders is necessary to address this complex public health problem.
JOURNALS OF GERONTOLOGY SERIES A-BIOLOGICAL SCIENCES AND MEDICAL SCIENCES
(2023)
Review
Genetics & Heredity
Valeria Graceffa
Summary: Although cross-correction shows potential in improving lysosomal storage diseases, cell-based therapies have faced difficulties and have not been successful. Advances in understanding lysosomal biology and gene therapy have led to increased research and progress in cell and gene therapy for LSDs.
CURRENT GENE THERAPY
(2022)
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
Martina Ricci, Gianpaolo Cicala, Anna Capasso, Giorgia Coratti, Stefania Fiori, Costanza Cutrona, Adele D'Amico, Valeria A. Sansone, Claudio Bruno, Sonia Messina, Tiziana Mongini, Michela Coccia, Gabriele Siciliano, Elena Pegoraro, Riccardo Masson, Massimiliano Filosto, Giacomo P. Comi, Stefania Corti, Dario Ronchi, Lorenzo Maggi, Maria G. D'Angelo, Veria Vacchiano, Chiara Ticci, Lucia Ruggiero, Lorenzo Verriello, Federica S. Ricci, Angela L. Berardinelli, Maria Antonietta Maioli, Matteo Garibaldi, Vincenzo Nigro, Stefano C. Previtali, Maria Carmela Pera, Eduardo Tizzano, Marika Pane, Francesco Danilo Tiziano, Eugenio Mercuri
Summary: This study provides an overview of the clinical phenotypes associated with 4 SMN2 copies. The results show variability in untreated patients, ranging from type II to type IV, with an overall reduction in functional scores as age increases. The risk of losing ambulation is significantly associated with SMA type, and women have a lower risk of losing ambulation compared to men.
ANNALS OF NEUROLOGY
(2023)
Review
Pediatrics
Panagiota Panagiotou, Christina Kanaka-Gantenbein, Athanasios G. Kaditis
Summary: Gene-based treatments do not have a significant positive impact on the ventilatory support requirements for SMA patients who are already on ventilatory support.
Review
Cell Biology
Andreas Rogalewski, Wolf-Ruediger Schaebitz
Summary: Stroke recovery processes involve restoration and compensation of lost or acquired functions. The translation of results from animal models to human situation is hindered by various factors, including genetic and anatomical differences, as well as differences in clinical function and behavior. The design of clinical trials also varies widely, making it difficult to compare and draw conclusions. Developing a recovery enhancing therapy for chronic stroke patients is a major unmet need in stroke research.
NEURAL REGENERATION RESEARCH
(2022)
Article
Environmental Studies
Gabriel da Silva Medina, Benno Pokorny
Summary: Agribusiness is an important development option for low-income countries, but the evidence of its positive effect on domestic agricultural sectors through foreign investments is unclear. The study on Brazil's agricultural development reveals high heterogeneity in the market share of domestic and multinational companies in key supply chains. In mixed segments, both foreign and domestic investments contribute to the optimization of supply chains, creating win-win situations and supporting the overall development of the industry.
Article
Clinical Neurology
Sarah J. Tabrizi, Carlos Estevez-Fraga, Willeke M. C. van Roon-Mom, Michael D. Flower, Rachael I. Scahill, Edward J. Wild, Ignacio Munoz-Sanjuan, Cristina Sampaio, Anne E. Rosser, Blair R. Leavitt
Summary: Huntington's disease is a prevalent neurodegenerative disorder with complex molecular pathogenesis. Currently, there is no effective treatment available. Potential interventions include targeting huntingtin DNA and RNA, clearing huntingtin protein, and DNA repair pathways. Evaluating past trials and considering the current situation will help in addressing the challenges and opportunities for the future.
Review
Genetics & Heredity
Michaela Drobek
Summary: Gene duplications lead to increased gene dosage, which can be retained or eliminated over time through functional diversification and the acquisition of novel functions. Functionally diverged paralogous genes can maintain some level of functional redundancy and partially replace each other. The gene dosage plays a crucial role in the development of specific tissues/organs, and alterations in gene dosage can have significant phenotypic consequences. The number of functional alleles, mutual cooperation, and interactions between paralogous genes influence the gene dosage and are important in development.
Review
Biochemical Research Methods
Brian Kelley, Pam De Moor, Kristen Douglas, Todd Renshaw, Stacey Traviglia
Summary: Several companies were authorized to treat COVID-19 patients with monoclonal antibodies within 1-2 years of the start of the pandemic. These products were developed and approved at an unprecedented speed, reducing clinical trial time by 75% or more without compromising safety. The therapies have successfully reduced hospitalization and death rates, benefiting hundreds of thousands of patients. The development strategies employed in this process have set a new precedent for speed, safety, and clinical benefit and are expected to have a lasting impact on future monoclonal antibody therapies.
CURRENT OPINION IN BIOTECHNOLOGY
(2022)
Review
Cell Biology
Anna Cazzola, Giovanni Cazzaniga, Andrea Biondi, Raffaella Meneveri, Silvia Brunelli, Emanuele Azzoni
Summary: Childhood leukemia, the most common cancer in young age, may originate during in utero development. Research on the cellular origin of leukemias is crucial for understanding disease progression, refining animal models, and developing new therapeutic approaches.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Review
Microbiology
Jack Adderley, Georges E. Grau
Summary: Host-directed therapies (HDT) are emerging as a new and effective approach to treat infectious diseases by inhibiting host factors necessary for pathogen development and strengthening host defense mechanisms. HDTs could be a promising strategy to combat drug resistance and provide protection against severe forms of diseases caused by malaria and other eukaryotic parasites through modulating the host immune response.
CURRENT OPINION IN MICROBIOLOGY
(2023)
Article
Clinical Neurology
Jiwon Lee, Se Eun Park, Dajeong Lee, Joo Young Song, Jeehun Lee
Summary: SMA type 1 is a severe condition with early onset and fast progression, often requiring permanent assisted ventilation. Nusinersen is currently the only approved treatment that can increase functional SMN protein levels. A case report demonstrates successful weaning from permanent ventilation with tracheostomy using nusinersen in an infant diagnosed with SMA type 1.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY
(2021)
