False positive findings associated with adenoviral vector-based vaccine underscore the regulatory necessity to eliminate abnormal toxicity test
In conclusion, ATT, which requires unnecessary animal usage and competes for resources which otherwise can be spent on innovative medicine research, should be deleted permanently as batch release test by regulatory authorities around the world.PMID:38561146 | DOI:10.1016/j.yrtph.2024.105617 (Source: Regulatory Toxicology and Pharmacology : RTP)
Source: Regulatory Toxicology and Pharmacology : RTP - April 1, 2024 Category: Toxicology Authors: Ryan Meng Mark van Ooij Yali Li Yunhai Zhang Jianxun Xie Source Type: research

State of Gene Therapy for Monogenic Cardiovascular Diseases
Over the past 2 decades, significant efforts have been made to advance gene therapy into clinical practice. Although successful examples exist in other fields, gene therapy for the treatment of monogenic cardiovascular diseases lags behind. In this review, we (1) highlight a brief history of gene therapy, (2) distinguish between gene silencing, gene replacement, and gene editing technologies, (3) discuss vector modalities used in the field with a special focus on adeno-associated viruses, (4) provide examples of gene therapy approaches in cardiomyopathies, channelopathies, and familial hypercholesterolemia, and (5) present...
Source: Mayo Clinic Proceedings - April 1, 2024 Category: Internal Medicine Authors: Sahej Bains, John R. Giudicessi, Katja E. Odening, Michael J. Ackerman Tags: Review Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research

DDX3X overexpression decreases dipeptide repeat proteins in a mouse model of C9ORF72-ALS/FTD
In this study, we examined the in vivo efficacy of DDX3X overexpression using a mouse model. We expressed exogenous DDX3X or GFP in the central nervous system (CNS) of the C9-500 ALS/FTD BAC transgenic or non-transgenic control mice using adeno-associated virus serotype 9 (AAV9). The DPR levels were significantly reduced in the brains of DDX3X-expressing C9-BAC mice compared to the GFP control even twelve months after virus delivery. Additionally, p62 aggregation was also decreased. No neuronal loss or neuroinflammatory response were detected in the DDX3X overexpressing C9-BAC mice. This work demonstrates that DDX3X overex...
Source: Experimental Neurology - March 31, 2024 Category: Neurology Authors: Xiujuan Fu Zhe Zhang Lindsey R Hayes Noelle Wright Julie Asbury Shelley Li Yingzhi Ye Shuying Sun Source Type: research

Assessing environmental exposure to viruses in wastewater treatment plant and swine farm scenarios with next-generation sequencing and occupational risk approaches
This study investigates the viral exposure of workers in a wastewater treatment plant (WWTP) and a swine farm by analyzing aerosol and surfaces samples. Viral contamination was evaluated using quantitative polymerase chain reaction (qPCR) assays, and target enrichment sequencing (TES) was performed to identify the vertebrate viruses to which workers might be exposed. Additionally, Quantitative Microbial Risk Assessment (QMRA) was conducted to estimate the occupational risk associated with viral exposure for WWTP workers, choosing Human Adenovirus (HAdV) as the reference pathogen. In the swine farm, QMRA was performed as an...
Source: International Journal of Hygiene and Environmental health - March 31, 2024 Category: Environmental Health Authors: Marta Itarte Miquel Calvo Lola Mart ínez-Frago Cristina Mej ías-Molina Sandra Mart ínez-Puchol Rosina Girones Gertjan Medema S ílvia Bofill-Mas Marta Rusi ñol Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research

DDX3X overexpression decreases dipeptide repeat proteins in a mouse model of C9ORF72-ALS/FTD
In this study, we examined the in vivo efficacy of DDX3X overexpression using a mouse model. We expressed exogenous DDX3X or GFP in the central nervous system (CNS) of the C9-500 ALS/FTD BAC transgenic or non-transgenic control mice using adeno-associated virus serotype 9 (AAV9). The DPR levels were significantly reduced in the brains of DDX3X-expressing C9-BAC mice compared to the GFP control even twelve months after virus delivery. Additionally, p62 aggregation was also decreased. No neuronal loss or neuroinflammatory response were detected in the DDX3X overexpressing C9-BAC mice. This work demonstrates that DDX3X overex...
Source: Experimental Neurology - March 31, 2024 Category: Neurology Authors: Xiujuan Fu Zhe Zhang Lindsey R Hayes Noelle Wright Julie Asbury Shelley Li Yingzhi Ye Shuying Sun Source Type: research

Assessing environmental exposure to viruses in wastewater treatment plant and swine farm scenarios with next-generation sequencing and occupational risk approaches
This study investigates the viral exposure of workers in a wastewater treatment plant (WWTP) and a swine farm by analyzing aerosol and surfaces samples. Viral contamination was evaluated using quantitative polymerase chain reaction (qPCR) assays, and target enrichment sequencing (TES) was performed to identify the vertebrate viruses to which workers might be exposed. Additionally, Quantitative Microbial Risk Assessment (QMRA) was conducted to estimate the occupational risk associated with viral exposure for WWTP workers, choosing Human Adenovirus (HAdV) as the reference pathogen. In the swine farm, QMRA was performed as an...
Source: International Journal of Hygiene and Environmental health - March 31, 2024 Category: Environmental Health Authors: Marta Itarte Miquel Calvo Lola Mart ínez-Frago Cristina Mej ías-Molina Sandra Mart ínez-Puchol Rosina Girones Gertjan Medema S ílvia Bofill-Mas Marta Rusi ñol Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research

DDX3X overexpression decreases dipeptide repeat proteins in a mouse model of C9ORF72-ALS/FTD
In this study, we examined the in vivo efficacy of DDX3X overexpression using a mouse model. We expressed exogenous DDX3X or GFP in the central nervous system (CNS) of the C9-500 ALS/FTD BAC transgenic or non-transgenic control mice using adeno-associated virus serotype 9 (AAV9). The DPR levels were significantly reduced in the brains of DDX3X-expressing C9-BAC mice compared to the GFP control even twelve months after virus delivery. Additionally, p62 aggregation was also decreased. No neuronal loss or neuroinflammatory response were detected in the DDX3X overexpressing C9-BAC mice. This work demonstrates that DDX3X overex...
Source: Experimental Neurology - March 31, 2024 Category: Neurology Authors: Xiujuan Fu Zhe Zhang Lindsey R Hayes Noelle Wright Julie Asbury Shelley Li Yingzhi Ye Shuying Sun Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research

DDX3X overexpression decreases dipeptide repeat proteins in a mouse model of C9ORF72-ALS/FTD
In this study, we examined the in vivo efficacy of DDX3X overexpression using a mouse model. We expressed exogenous DDX3X or GFP in the central nervous system (CNS) of the C9-500 ALS/FTD BAC transgenic or non-transgenic control mice using adeno-associated virus serotype 9 (AAV9). The DPR levels were significantly reduced in the brains of DDX3X-expressing C9-BAC mice compared to the GFP control even twelve months after virus delivery. Additionally, p62 aggregation was also decreased. No neuronal loss or neuroinflammatory response were detected in the DDX3X overexpressing C9-BAC mice. This work demonstrates that DDX3X overex...
Source: Experimental Neurology - March 31, 2024 Category: Neurology Authors: Xiujuan Fu Zhe Zhang Lindsey R Hayes Noelle Wright Julie Asbury Shelley Li Yingzhi Ye Shuying Sun Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research

DDX3X overexpression decreases dipeptide repeat proteins in a mouse model of C9ORF72-ALS/FTD
In this study, we examined the in vivo efficacy of DDX3X overexpression using a mouse model. We expressed exogenous DDX3X or GFP in the central nervous system (CNS) of the C9-500 ALS/FTD BAC transgenic or non-transgenic control mice using adeno-associated virus serotype 9 (AAV9). The DPR levels were significantly reduced in the brains of DDX3X-expressing C9-BAC mice compared to the GFP control even twelve months after virus delivery. Additionally, p62 aggregation was also decreased. No neuronal loss or neuroinflammatory response were detected in the DDX3X overexpressing C9-BAC mice. This work demonstrates that DDX3X overex...
Source: Experimental Neurology - March 31, 2024 Category: Neurology Authors: Xiujuan Fu Zhe Zhang Lindsey R Hayes Noelle Wright Julie Asbury Shelley Li Yingzhi Ye Shuying Sun Source Type: research

B-Type Natriuretic Peptide Inhibits the Expression and Function of SERCA2a in Heart Failure
Int Heart J. 2024;65(2):292-299. doi: 10.1536/ihj.23-144.ABSTRACTB-type natriuretic peptide (BNP) possesses protective cardiovascular properties; however, there has not been sufficient serious consideration of the side effects of BNP. As for sarcoplasmic/endoplasmic reticulum calcium ATPase 2a (SERCA2a), it was once considered a new target for the treatment of heart failure (HF). Nevertheless, clinical trials of SERCA2a gene therapy in HF have finally become unsuccessful. Research has found that elevated BNP levels and decreased SERCA2a expression are two important HF characteristics, which are always negatively correlated...
Source: International Heart Journal - March 31, 2024 Category: Cardiology Authors: Yuting Zhai Junhong Chen Rongsheng Kan Haochen Xuan Chaofan Wang Dongye Li Tongda Xu Source Type: research