Article
Engineering, Biomedical
Sikai Ling, Shiqi Yang, Xinde Hu, Di Yin, Yao Dai, Xiaoqing Qian, Dawei Wang, Xiaoyong Pan, Jiaxu Hong, Xiaodong Sun, Hui Yang, Soren Riis Paludan, Yujia Cai
Summary: The study demonstrates the efficacy of engineered lentiviruses co-packaging SpCas9 mRNA and a guide RNA targeting the Vegfa gene in preventing wet age-related macular degeneration in mice, providing a potential new treatment approach for retinal neovascular diseases.
NATURE BIOMEDICAL ENGINEERING
(2021)
Article
Cell Biology
Xuejiao Yuan, Yanfeng Zhou, Jinli Sun, Shanshan Wang, Xingjie Hu, Jiyu Li, Jing Huang, Nan Chen
Summary: Acute liver injury (ALI) is a severe liver disease characterized by sudden hepatocyte necrosis and liver function deterioration. This study develops hepatocyte-targeting self-assembling nanoparticles encapsulating an organic Selenium compound, which effectively removes reactive oxygen species (ROS) and protects hepatocyte viability. The nanoparticles, functionalized with a liver-targeting ligand, exhibit enhanced hepatocyte uptake and successfully reduce liver damage in mouse models of ALI induced by acetaminophen or carbon tetrachloride.
CELL PROLIFERATION
(2023)
Article
Pharmacology & Pharmacy
Marina Cortijo-Gutierrez, Sabina Sanchez-Hernandez, Maria Tristan-Manzano, Noelia Maldonado-Perez, Lourdes Lopez-Onieva, Pedro J. Real, Concha Herrera, Juan Antonio Marchal, Francisco Martin, Karim Benabdellah
Summary: The incorporation of IS2 into IDLVs improves both transgene expression levels and their ability to be inserted into existing double strand breaks (DSBs), offering potential implications for the development of an unbiased detection tool for off-target cleavage sites from different specific nucleases.
Review
Pharmacology & Pharmacy
Yvan Arsenijevic, Adeline Berger, Florian Udry, Corinne Kostic
Summary: This review provides an overview of lentiviral vector technologies, their application in ophthalmology, and their potential for gene therapy.
Review
Pharmacology & Pharmacy
Rama Kashikar, Arun K. Kotha, Saurabh Shah, Paras Famta, Shashi Bala Singh, Saurabh Srivastava, Mahavir Bhupal Chougule
Summary: RNA binding proteins play a crucial role in RNA regulation, and their abnormal expression can lead to disease progression. This article discusses the latest findings in cancer therapy and diagnosis targeting RNA binding proteins, as well as the application of various interventions and the development of nanoparticle delivery technologies.
ADVANCED DRUG DELIVERY REVIEWS
(2022)
Review
Pharmacology & Pharmacy
Rama Kashikar, Arun K. Kotha, Saurabh Shah, Paras Famta, Shashi Bala Singh, Saurabh Srivastava, Mahavir Bhupal Chougule
Summary: RNA binding proteins play a crucial role in RNA metabolism and their dysregulation can lead to diseases. This article discusses interventions and nanoparticles used for cancer therapy and diagnosis by targeting RNA binding proteins.
ADVANCED DRUG DELIVERY REVIEWS
(2022)
Article
Medicine, Research & Experimental
John R. Counsell, Guillaume De Brabandere, Rajvinder Karda, Marc Moore, Antonio Greco, Alysha Bray, Juan Antinao Diaz, Dany P. Perocheau, Ulrike Mock, Simon N. Waddington
Summary: Optimization of a reverse-transcriptase-deficient LV vector enables direct translation of LV RNA genomes upon cell entry, transiently expressing vector payloads as mRNA without a DNA intermediate. Engineering a novel LV genome arrangement removes HIV-1 sequences from the 5' end, allowing efficient translation of the vector payload through ribosomal entry from the 5' 7-methylguanylate cap. LV-mediated mRNA delivery platform provides temporary transgene expression in vitro and in vivo for potential applications in gene and cell therapy scenarios.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2021)
Article
Virology
Maira Zorzan, Claudia Del Vecchio, Stefania Vogiatzis, Elisa Saccon, Cristina Parolin, Giorgio Palu, Arianna Calistri, Carla Mucignat-Caretta
Summary: In this study, a third-generation lentiviral system was developed to deliver shRNA targeting the regulatory subunit R2Alpha of protein kinase A, resulting in efficient and stable downregulation of R2Alpha in glioblastoma cells. The silencing of R2Alpha was found to potentially reduce the viability of tumor cells, highlighting the promising role of protein kinase A as a target for novel anti-glioma therapies.
Article
Virology
Aitthiphon Chongchai, Sajee Waramit, Tunchanok Wongwichai, Jirawan Kampangtip, Thanyaluck Phitak, Prachya Kongtawelert, Amin Hajitou, Keittisak Suwan, Peraphan Pothacharoen
Summary: The study developed a targeted delivery strategy which successfully delivered transgenes to HACs using CAP.Phage particles, with the CAP ligand demonstrating preference for binding to pathogenic chondrocytes.
Review
Biochemistry & Molecular Biology
Zahra Zahed, Raha Hadi, Gholamhassan Imanzadeh, Zainab Ahmadian, Sasan Shafiei, Amin Zaki Zadeh, Hanie Karimi, Amirhossein Akbarzadeh, Mahmoud Abbaszadeh, Laleh Saleh Ghadimi, Hossein Samadi Kafil, Fahimeh Kazeminava
Summary: This article reviews the latest research developments in gene delivery, with a focus on the potential of quantum dots as a tool for gene delivery. By evaluating the physicochemical characteristics of quantum dots, researchers believe that they can be used in biosensing imaging, drug delivery, and gene delivery applications.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2024)
Article
Biochemistry & Molecular Biology
Jing Yang, Tetsuya Hirata, Yi-Shi Liu, Xin-Yu Guo, Xiao-Dong Gao, Taroh Kinoshita, Morihisa Fujita
Summary: Many GPI-APs like CD59, CD55, and CD109 utilize the hSND2-dependent ER targeting machinery, with signal recognition particle receptors cooperating to facilitate this process. The hydrophobicity of the C-terminal GPI attachment signal serves as a determinant of hSND2 dependency in ER targeting of GPI-APs.
Article
Cardiac & Cardiovascular Systems
Yasushi Konegawa, Toshie Kuwahara, Jun-Ichiro Jo, Kozue Murata, Takahide Takeda, Tadashi Ikeda, Kenji Minatoya, Hidetoshi Masumoto, Yasuhiko Tabata
Summary: In this study, the therapeutic effects and mechanisms of Pioglitazone (PGZ) were validated using a drug delivery system. The results showed that PGZ-MS could improve cardiac dysfunction and reverse ventricular remodeling by enhancing macrophage polarization towards the M2 subtype and attenuating cardiomyocyte apoptosis.
EUROPEAN JOURNAL OF CARDIO-THORACIC SURGERY
(2022)
Article
Multidisciplinary Sciences
Helin Sertkaya, Mattia Ficarelli, Nathan P. Sweeney, Hannah Parker, Conrad A. Vink, Chad M. Swanson
Summary: Many lentiviral vectors for gene therapy are derived from HIV-1, and through systematic deletion analysis, it has been found that significant portions of the gag and env regions can be removed without compromising transduction efficiency. Long-read sequencing has also been utilized to study splice site usage, showing that specific deletions in the env sequence can reduce splicing events per transcript. These findings allow for the creation of lentiviral vectors with minimal HIV-1 sequence, potentially improving safety and reducing viral sequence transfer to patients' DNA.
SCIENTIFIC REPORTS
(2021)
Article
Biochemistry & Molecular Biology
Yanira Zeyn, Dominika Hobernik, Ulrich Wilk, Jana Poehmerer, Christoph Hieber, Carolina Medina-Montano, Nadine Roehrig, Caroline F. Straehle, Andrea K. Thoma-Kress, Ernst Wagner, Matthias Bros, Simone Berger
Summary: A deeper understanding of the role of the tumor microenvironment in cancer development has led to the development of new strategies, such as gene-based cancer immunotherapy. This study tested the concept of DC-focused transcriptional targeting by modifying and optimizing plasmids to achieve specific gene expression in antigen-presenting cells.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Medicine, Research & Experimental
Angela H. Braun, Annika M. Frank, Naphang Ho, Christian J. Buchholz
Summary: CD3-targeted lentiviral vectors (CD3-LVs) effectively transduce human T lymphocytes and activate them. The use of Src/Abl tyrosine kinase inhibitor dasatinib enhances gene delivery by T cell-targeted LVs. Dasatinib treatment prior to incubation with CD3-LV improves reporter gene delivery and transduction into non-activated target cells.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
(2023)
Article
Multidisciplinary Sciences
Juliette Simonin, Emmanuelle Bille, Gilles Crambert, Sabrina Noel, Elise Dreano, Aurelie Edwards, Aurelie Hatton, Iwona Pranke, Berengere Villeret, Charles-Henry Cottart, Jean-Patrick Vrel, Valerie Urbach, Nesrine Baatallah, Alexandre Hinzpeter, Anita Golec, Lhousseine Touqui, Xavier Nassif, Luis J. V. Galietta, Gabrielle Planelles, Jean-Michel Sallenave, Aleksander Edelman, Isabelle Sermet-Gaudelus
SCIENTIFIC REPORTS
(2019)
Article
Biochemistry & Molecular Biology
Sebastien Gauthier, Iwona Pranke, Vincent Jung, Loredana Martignetti, Veronique Stoven, Thao Nguyen-Khoa, Michaela Semeraro, Alexandre Hinzpeter, Aleksander Edelman, Ida Chiara Guerrera, Isabelle Sermet-Gaudelus
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2020)
Article
Multidisciplinary Sciences
Sara Bitam, Ahmad Elbahnsi, Geordie Creste, Iwona Pranke, Benoit Chevalier, Farouk Berhal, Brice Hoffmann, Nathalie Servel, Danielle Tondelier, Aurelie Hatton, Christelle Moquereau, Melanie Faria Da Cunha, Alexandra Pastor, Agathe Lepissier, Alexandre Hinzpeter, Jean-Paul Mornon, Guillaume Prestat, Aleksander Edelman, Isabelle Callebaut, Christine Gravier-Pelletier, Isabelle Sermet-Gaudelus
Summary: The compound C407 and its derivatives correct the F508del-CFTR protein by stabilizing specific positions on the protein molecule. An analog of C407, G1, significantly improves CFTR activity by interacting more effectively with ICL4. These findings suggest new strategies for optimizing the ICL4-NBD1 interface.
SCIENTIFIC REPORTS
(2021)
Correction
Multidisciplinary Sciences
Sara Bitam, Ahmad Elbahnsi, Geordie Creste, Iwona Pranke, Benoit Chevalier, Farouk Berhal, Brice Hoffmann, Nathalie Servel, Nesrine Baatalah, Danielle Tondelier, Aurelie Hatton, Christelle Moquereau, Melanie Faria Da Cunha, Alexandra Pastor, Agathe Lepissier, Alexandre Hinzpeter, Jean-Paul Mornon, Guillaume Prestat, Aleksander Edelman, Isabelle Callebaut, Christine Gravier-Pelletier, Isabelle Sermet-Gaudelus
SCIENTIFIC REPORTS
(2021)
Article
Biochemistry & Molecular Biology
Nesrine Baatallah, Ahmad Elbahnsi, Jean-Paul Mornon, Benoit Chevalier, Iwona Pranke, Nathalie Servel, Renaud Zelli, Jean-Luc Decout, Aleksander Edelman, Isabelle Sermet-Gaudelus, Isabelle Callebaut, Alexandre Hinzpeter
Summary: Protein misfolding is associated with various diseases, including cystic fibrosis. Correctors like VX-809, VX-661, and VX-445 have been developed to rescue mutant proteins; through blind docking and molecular dynamics simulations, potential binding sites and mechanisms of action have been identified. These correctors stabilize protein-lipid interfaces and enhance inter-domain assembly, providing novel insights into rescuing misfolded proteins with small molecules.
CELLULAR AND MOLECULAR LIFE SCIENCES
(2021)
Article
Respiratory System
Aurelie Hatton, Anne Bergougnoux, Katarzyna Zybert, Benoit Chevalier, Myriam Mesbahi, Jean Pierre Alteri, Katarzyna Walicka-Serzysko, Magdalena Postek, Magali Taulan-Cadars, Aleksander Edelman, Alexandre Hinzpeter, Mireille Claustres, Emmanuelle Girodon, Caroline Raynal, Isabelle Sermet-Gaudelus, Dorota Sands, Dorota Sands
Summary: This study demonstrates the usefulness of combining genetic and functional investigations to assess the possibility of evolving to CF or CFTR-RD in babies with inconclusive diagnosis at neonatal screening.
JOURNAL OF CYSTIC FIBROSIS
(2022)
Article
Neurosciences
Sabrina Noel, Nathalie Servel, Aurelie Hatton, Anita Golec, Mayuree Rodrat, Demi R. S. Ng, Hongyu Li, Iwona Pranke, Alexandre Hinzpeter, Aleksander Edelman, David N. Sheppard, Isabelle Sermet-Gaudelus
Summary: This study investigates the relationship between genotype, phenotype, and CFTR function using human nasal epithelial cells, revealing differences in CFTR-mediated currents based on residual or minimal CFTR function. The findings suggest that CFTR expression and function are not affected by conditional reprogramming culture, and highlight the correlation between CFTR-mediated Cl- currents in hNE cells and sweat Cl- concentration, emphasizing the value of these cells in CFTR research and therapeutic testing.
JOURNAL OF PHYSIOLOGY-LONDON
(2022)
Article
Multidisciplinary Sciences
Melanie Faria da Cunha, Iwona Pranke, Ali Sassi, Christiane Schreiweis, Stephanie Moriceau, Dragana Vidovic, Aurelie Hatton, Mariane Sylvia Carlon, Geordie Creste, Farouk Berhal, Guillaume Prestat, Romain Freund, Norbert Odolczyk, Jean Philippe Jais, Christine Gravier-Pelletier, Piotr Zielenkiewicz, Vincent Jullien, Alexandre Hinzpeter, Franck Oury, Aleksander Edelman, Isabelle Sermet-Gaudelus
Summary: This study provides the first in vivo preclinical evidence of the efficacy of c407 in treating Cystic Fibrosis, correcting CFTR dysfunction and improving chloride conductance. Importantly, c407 was well tolerated and demonstrated no obvious toxicity after systemic administration.
SCIENTIFIC REPORTS
(2022)
Article
Biochemistry & Molecular Biology
Benoit Chevalier, Nesrine Baatallah, Matthieu Najm, Solene Castanier, Vincent Jung, Iwona Pranke, Anita Golec, Veronique Stoven, Stefano Marullo, Fabrice Antigny, Ida Chiara Guerrera, Isabelle Sermet-Gaudelus, Aleksander Edelman, Alexandre Hinzpeter
Summary: In this study, the interaction of CFTR and its mutants with other proteins was investigated using various experimental approaches. Proximity labeling methods based on TurboID and APEX2 were evaluated and compared to existing databases. The results revealed new protein partners and provided a more comprehensive understanding of the CFTR interactome.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
Kusumika Saha, Benoit Chevalier, Stephane Doly, Nesrine Baatallah, Thomas Guilbert, Iwona Pranke, Mark G. H. Scott, Herve Enslen, Chiara Guerrera, Cerina Chuon, Aleksander Edelman, Isabelle Sermet-Gaudelus, Alexandre Hinzpeter, Stefano Marullo
Summary: The exit of certain plasma membrane proteins from the endoplasmic reticulum is regulated by arginine-based retention motifs. PRAF2, a gatekeeper protein, recognizes these motifs and retains specific proteins in the ER. It has been found that PRAF2 can interact with mutant CFTR, preventing it from reaching the ER exit sites. Pharmacological chaperones have also been shown to rescue CFTR-F508del retention by PRAF2 through various mechanisms. These findings provide new therapeutic perspectives for diseases caused by impaired cell surface trafficking of mutant proteins.
CELLULAR AND MOLECULAR LIFE SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
Iwona Maria Pranke, Benoit Chevalier, Aiswarya Premchandar, Nesrine Baatallah, Kamil F. Tomaszewski, Sara Bitam, Danielle Tondelier, Anita Golec, Jan Stolk, Gergely L. Lukacs, Pieter S. Hiemstra, Michal Dadlez, David A. Lomas, James A. Irving, Agnes Delaunay-Moisan, Eelco van Anken, Alexandre Hinzpeter, Isabelle Sermet-Gaudelus, Aleksander Edelman
Summary: Early recognition and enhanced degradation of misfolded proteins by the endoplasmic reticulum (ER) quality control and ER-associated degradation (ERAD) are important mechanisms. This study found that decreasing Keratin 8 (K8) expression increased the secretion of misfolded Z-alpha-1-antitrypsin (Z-A1AT) protein and F508del-CFTR protein. K8 was shown to regulate the Hrd1-governed ERAD pathway and may act as a scaffolding protein in ERAD complexes. Targeting K8-containing ERAD complexes could be a promising strategy for the treatment of alpha-1-antitrypsin deficiency (A1ATD).
CELLULAR AND MOLECULAR LIFE SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
Nesrine Baatallah, Ahmad Elbahnsi, Benoit Chevalier, Solene Castanier, Jean-Paul Mornon, Iwona Pranke, Aleksander Edelman, Isabelle Sermet-Gaudelus, Isabelle Callebaut, Alexandre Hinzpeter
Summary: A second-site mutation near the CFTR MSD1 groove partially rescued the folding defect caused by cystic fibrosis mutations in MSD1, providing insights into the key features of folding and function of the CFTR protein and type IV ABC transporters in general.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2022)
Article
Biochemistry & Molecular Biology
Marc Ravatin, Norbert Odolczyk, Nathalie Servel, J. Inaki Guijarro, Eric Tagat, Benoit Chevalier, Nesrine Baatallah, Pierre-Jean Corringer, Gergely L. Lukacs, Aleksander Edelman, Piotr Zielenkiewicz, Jean -Marie Chambard, Alexandre Hinzpeter, Grazyna Faure
Summary: The study used a structure-based computational approach to design peptides that mimic the CBb-AF508NBD1 interface. These peptides enhance chloride channel activity and correct the trafficking defect of misfolded AF508CFTR. They bind to the same region as CBb phospholipase A2 on AF508NBD1 and show synergy with the clinically approved VX-770 potentiator. These findings contribute to the development of new therapeutics for cystic fibrosis.
JOURNAL OF MOLECULAR BIOLOGY
(2023)
Letter
Biochemistry & Molecular Biology
Mickaeel Lhuillier, Laura Aoust, Elise Dreano, Marie-Laure Franco-Montoya, Kim Landry-Truchon, Nicolas Houde, Stephanie Chhun, Alexandre Hinzpeter, Aleksander Edelman, Christophe Delacourt, Lucie Jeannotte, Isabelle Sermet-Gaudelus, Alice Hadchouel
AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY
(2022)
Article
Respiratory System
Aurelie Hatton, Anne Bergougnoux, Katarzyna Zybert, Benoit Chevalier, Myriam Mesbahi, Jean Pierre Alteri, Katarzyna Walicka-Serzysko, Magdalena Postek, Magali Taulan-Cadars, Aleksander Edelman, Alexandre Hinzpeter, Mireille Claustres, Emmanuelle Girodon, Caroline Raynal, Isabelle Sermet-Gaudelus, Dorota Sands
Summary: This study demonstrates the usefulness of combining genetic and functional investigations to assess the possibility of evolving to CF or CFTR-RD in babies with inconclusive diagnosis at neonatal screening.
JOURNAL OF CYSTIC FIBROSIS
(2022)