Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
Gene therapy for neurodegenerative disorders in children: dreams and realities
Arch Pediatr. 2023 Nov;30(8S1):8S32-8S40. doi: 10.1016/S0929-693X(23)00225-7.ABSTRACTGene therapy encompasses the administration of biological medicinal products containing recombinant nucleic acids, mainly DNA, with the aim of treating or curing diseases. This represents a unique therapeutic strategy to reach the brain, in order to prevent or halt a neurodegenerative process. During the past decade, active multidisciplinary research has started to solve many issues for gene therapy in neurodegenerative disorders in terms of vectors, modes of administration, and expression of the therapeutic DNA. The engineering of hematop...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Odile Boespflug-Tanguy Caroline Sevin Francoise Piguet Source Type: research
Gene therapy in spinal muscular atrophy
Arch Pediatr. 2023 Nov;30(8S1):8S12-8S17. doi: 10.1016/S0929-693X(23)00222-1.ABSTRACTInfantile SMA is a neuromuscular disease caused by the motor neuron degeneration, depending on the age of appearance of clinical signs and the evolution of the disease, three types of decreasing severity have been defined. SMA is caused by mutations or deletions of the SMN1 gene and disease. Various therapies aimed at increasing SMN protein levels have been developed. Gene therapy is part of the therapeutic arsenal now available for the treatment of SMA under certain conditions. It uses the scAAV9 vector carrying a functional copy of SMN1 ...
Source: Archives de Pediatrie - December 3, 2023 Category: Pediatrics Authors: Fr édérique Audic Source Type: research
