Results 111 to 120 of about 12,497,887 (407)

Cancer cell death induced by the NAD antimetabolite Vacor discloses the antitumor potential of SARM1

FEBS Letters, EarlyView.
Vacor, a compound converted into the toxic metabolite Vacor adenine dinucleotide (VAD) by the nicotinamide salvage pathway enzymes NAMPT and NMNAT2, exhibits antitumor activity by inducing rapid and complete NAD depletion. We report that Vacor toxicity is limited to cell lines expressing high levels of SARM1, a NAD glycohydrolase.
Giuseppe Ranieri, Andrea Lapucci, Giuseppe Orsomando, Nadia Raffaelli, Alberto Chiarugi, Daniela Buonvicino   +5 more
wiley   +1 more source

Gene Therapy for Hemoglobinopathies [PDF]

Human Gene Therapy, 2018
Gene therapy for β-thalassemia and sickle-cell disease is based on transplantation of genetically corrected, autologous hematopoietic stem cells. Preclinical and clinical studies have shown the safety and efficacy of this therapeutic approach, currently based on lentiviral vectors to transfer a β-globin gene under the transcriptional control of ...
Cavazzana M., Mavilio F.
openaire   +3 more sources

RAD50 missense variants differentially affect the DNA damage response and mitotic progression

FEBS Letters, EarlyView.
RAD50 incorporates into the MRN complex and initiates the DNA damage response. Furthermore, RAD50 promotes mitotic progression. RAD50 missense variants capable of forming an MRN complex supported the DNA damage response and mitotic features to different extents in complementation experiments, indicating these functions are separable and might impact ...
Hanna Redeker, Swantje Kebel, Lea Völkening, Anna Vatselia, Louisa Weinhold, Girmay Asgedom, Axel Schambach, Detlev Schindler, Thilo Dörk, Kristine Bousset   +9 more
wiley   +1 more source

Enteropathogenic E. coli shows delayed attachment and host response in human jejunum organoid‐derived monolayers compared to HeLa cells

FEBS Letters, EarlyView.
Enteropathogenic E. coli (EPEC) infects the human intestinal epithelium, resulting in severe illness and diarrhoea. In this study, we compared the infection of cancer‐derived cell lines with human organoid‐derived models of the small intestine. We observed a delayed in attachment, inflammation and cell death on primary cells, indicating that host ...
Mastura Neyazi, Pilar Samperio Ventayol, Natalie Burkard, Nicolas Schlegel, Carmen Aguilar, Sina Bartfeld   +5 more
wiley   +1 more source

Involvement of hepatic macrophages in the antifibrotic effect of IGF-I-overexpressing mesenchymal stromal cells

Stem Cell Research & Therapy, 2016
Background Cirrhosis is a major health problem worldwide and new therapies are needed. Hepatic macrophages (hMø) have a pivotal role in liver fibrosis, being able to act in both its promotion and its resolution.
Esteban Fiore, Mariana Malvicini, Juan Bayo, Estanislao Peixoto, Catalina Atorrasagasti, Romina Sierra, Marcelo Rodríguez, Sofia Gómez Bustillo, Mariana G. García, Jorge B. Aquino, Guillermo Mazzolini   +10 more
doaj   +1 more source

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

BioDrugs, 2017
There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells ...
M. Naso, B. Tomkowicz, William L Perry, W. Strohl   +3 more
semanticscholar   +1 more source

Non-viral approaches to gene therapy [PDF]

, 1993
Several advances in non-viral gene transfer technology have been reported over the past year. Cationic lipids have been successfully used to deliver genes in vivo, providing a clear alternative to recombinant viruses.
Behr, Behr, Blumenthal, Böttger, Cotten, Cotten, Cotten, Cristiano, Curiel, Demeneix, Ellens, Felgner, Felgner, Fitzgerald, Haensler, Haensler, Hawley-Nelson, Huckett, Hyde, Kaneda, Legendre, Leonetri, Midoux, Nabel, Parente, Plank, Plautz, Rosenkranz, Seth, Shen, Stribling, Subbarao, Tang, Tomita, Trubetskoy, Trubetskoy, Ulmer, Wagner, Wagner, Wagner, Wagner, Wagner, Williams, Wu, Zhu   +44 more
core   +1 more source

4‐nitrobenzoate inhibits 4‐hydroxybenzoate polyprenyltransferase in malaria parasites and enhances atovaquone efficacy

FEBS Letters, EarlyView.
Atovaquone is an antimalarial requiring potentiation for sufficient efficacy. We pursued strategies to enhance its activity, showing that 4‐nitrobenzoate inhibits 4‐hydroxybenzoate polyprenyltransferase, decreasing ubiquinone biosynthesis. Since atovaquone competes with ubiquinol in mitochondria, 4‐nitrobenzoate facilitates its action, potentiating ...
Ignasi Bofill Verdaguer, Matheus Felipe Santos, Maurício Mazzine Filho, Gabriela Oliveira Castro, Agustín Hernández, Manoel Aparecido Peres, Alejandro Miguel Katzin, Marcell Crispim   +7 more
wiley   +1 more source

Clinical and molecular features and therapeutic perspectives of spinal muscular atrophy with respiratory distress type 1 [PDF]

, 2015
Spinal muscular atrophy with respiratory distress (SMARD1) is an autosomal recessive neuromuscular disease caused by mutations in the IGHMBP2 gene, encoding the immunoglobulin μ-binding protein 2, leading to motor neuron degeneration.
Corti, Stefania, Parente, Valeria, Porro, Francesca, Rinchetti, Paola, Vanoli, Fiammetta   +4 more
core   +2 more sources

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source