يعرض 1 - 20 نتائج من 36 نتيجة بحث عن '"Terapias de RNA"', وقت الاستعلام: 0.94s تنقيح النتائج
  1. 1
    Paving the Road for RNA Therapeutics: Focus on Lipid-based Nanocarriers for mRNA Delivery
    Preparar o Caminho para as Terapias de RNA: Foco em Nanotransportadores baseados em Lípidos para a Entrega de mRNA

    المؤلفون: Henriques, Carolina Nunes Bebiano

    مصطلحات موضوعية: Terapias de RNA, Tecnologias de Entrega de RNA, mRNA, Nanopartículas Lipídicas, QbD, RNA Therapeutics, RNA Delivery Technologies, Lipid Nanoparticles

    الاتاحة: https://hdl.handle.net/10316/105571

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  2. 2
    Targeted RNA-based therapies for Mucopolysaccharidosis

    المؤلفون: Santos, Juliana Inês, Gonçalves, Mariana, Matos, Liliana, Gaspar, Paulo, Pires, Maria João, Oliveira, Paula, Prata, Maria João, Coutinho, Maria Francisca, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Mucopolissacaridoses, Terapias de RNA, Lysosomal Storage Disorders, RNA Therapeutics

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/8198

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  3. 3
    RNA como alvo terapêutico: desenvolvimento de abordagens inovadoras utilizando oligonucleótidos sintéticos.

    المؤلفون: Matos, Liliana, Coutinho, Maria Francisca, Santos, Juliana Inês, Gonçalves, Mariana, Duarte, Ana Joana, Ribeiro, Diogo, Moreira, Luciana, Encarnação, Marisa, Gaspar, Paulo, Amaral, Olga, Carvalho, Sofia, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Terapias de RNA, Doenças Lisossomais de Sobrecarga

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/8196

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  4. 4
    Antisense oligonucleotide exon-skipping as a therapeutic approach for Mucolipidosis type II α/β: in vitro and in vivo studies

    المؤلفون: Gonçalves, Mariana, Matos, Liliana, Santos, Juliana Inês, Coutinho, Maria Francisca, Prata, Maria João, Pires, Maria João, Oliveira, Paula, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Doenças Lisossomais de Sobrecarga, Terapias de RNA, Mucolipidose tipo II

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/8189

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  5. 5
    When less is actually more: in vitro assessment of the potential of anti-XYLT1 siRNAs to promote substrate reduction in Mucopolysaccharidosis type III

    المؤلفون: Santos, Juliana Inês, Coutinho, Maria Francisca, Gaspar, Paulo, Prata, Maria João, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Mucopolissacaridose III, Doenças Lisossomais de sobrecarga, Terapias de RNA, Genética Humana, Doenças Genéticas

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/8144

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  6. 6
    Mutation-adapted U1snRNA as a therapeutic strategy for Mucopolysaccharidosis IIIC: in vitro and in vivo studies

    المؤلفون: Gonçalves, Mariana, Matos, Liliana, Santos, Juliana Inês, Coutinho, Maria Francisca, Prata, Maria João, Pires, Maria João, Oliveira, Paula, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Doenças Lisossomais de Sobrecarga, Terapias de RNA, MPS IIIC

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/8194

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  7. 7
    Conference
    When less is actually more: in vitro assessment of the potential of anti-XYLT1 siRNAs to promote substrate reduction in Mucopolysaccharidosis type III

    المؤلفون: Santos, Juliana Inês, Coutinho, Maria Francisca, Gaspar, Paulo, Prata, Maria João, Alves, Sandra

    مصطلحات موضوعية: Mucopolissacaridose III, Doenças Lisossomais de sobrecarga, Terapias de RNA, Genética Humana, Doenças Genéticas

    Relation: info:eu-repo/grantAgreement/FCT/OE/SFRH%2FBPD%2F101965%2F2014/PT; info:eu-repo/grantAgreement/FCT/OE/SFRH%2FBD%2F124372%2F2016/PT; http://hdl.handle.net/10400.18/8144

    الاتاحة: http://hdl.handle.net/10400.18/8144

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  8. 8
    Conference
    Targeted RNA-based therapies for Mucopolysaccharidosis

    المؤلفون: Santos, Juliana Inês, Gonçalves, Mariana, Matos, Liliana, Gaspar, Paulo, Pires, Maria João, Oliveira, Paula, Prata, Maria João, Coutinho, Maria Francisca, Alves, Sandra

    مصطلحات موضوعية: Mucopolissacaridoses, Doenças Lisossomais de Sobrecarga, Terapias de RNA, Genética Humana, Doenças Genéticas

    Relation: http://hdl.handle.net/10400.18/8146

    الاتاحة: http://hdl.handle.net/10400.18/8146

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  9. 9
    Conference
    Antisense oligonucleotide exon-skipping as a therapeutic approach for Mucolipidosis type II α/β: in vitro and in vivo studies

    المؤلفون: Gonçalves, Mariana, Matos, Liliana, Santos, Juliana Inês, Coutinho, Maria Francisca, Prata, Maria João, Pires, Maria João, Oliveira, Paula, Alves, Sandra

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Doenças Lisossomais de Sobrecarga, Terapias de RNA, Mucolipidose tipo II

    Relation: http://hdl.handle.net/10400.18/8189

    الاتاحة: http://hdl.handle.net/10400.18/8189

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  10. 10
    From bench to bioterium and back again: development of a U1snRNA-based therapeutic strategy for mucopolysaccharidosis IIIC

    المؤلفون: Santos, Juliana Inês, Matos, Liliana, Oliveira, Paula Alexandra, Gonçalves, Mariana, Pires, Maria João, Coutinho, Maria Francisca, Prata, Maria João, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Lysosomal Storage Diseases, RNA Therapies, Doenças Lisossomais de sobrecarga, Doenças Genéticas, Terapias de RNA

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/7462

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  11. 11
    From bench to bioterium and back again: development of a U1snRNA-based therapeutic strategy for mucopolysaccharidosis IIIC

    المؤلفون: Santos, Juliana Inês Pinto, Matos, Liliana, Oliveira, Paula Alexandra, Gonçalves, Mariana, Pires, Maria João, Coutinho, Maria Francisca, Prata, Maria João, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Lysosomal Storage Diseases, RNA Therapies, Doenças Lisossomais de sobrecarga, Terapias de RNA, Doenças Genéticas, Mucopolysaccharidoses type III

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/7457

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  12. 12
    The 2020s tooth fairy: from loose tooth to neuronal cell cultures, a method to establish patient-derived MPS III neuronal cell lines

    المؤلفون: Coutinho, Maria Francisca, Santos, Juliana Inês, Matos, Liliana, Alves, Sandra

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Lysosomal Storage Diseases, MPS III, Therapeutic RNA Drugs, Dental Pulp Stem Cells, Patient-derived MPS III Neuronal Cell Lines, Doenças Genéticas, Terapias de RNA

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/7461

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  13. 13
    The use of a modified U1 snRNA as a therapeutic strategy to correct a 5’ splice-site mutation in Mucopolysaccharidosis IIIC: in vitro steps towards an in vivo approach

    المؤلفون: Matos, L., Santos, J.I., Rocha, M., Coutinho, M.F., Prata, M.J., Alves, S.

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Lysosomal Storage Disorders, RNA Therapies, Terapias de RNA, Terapias de Splicing, U1 snRNA, MPS IIIC, Doenças Lisossomais de Sobrecarga, Doenças Genéticas

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/6792

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  14. 14
    Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

    المؤلفون: Matos, L., Santos, J.I., Rocha, M., Coutinho, M.F., Gaspar, P., Voltolini Velho, R., Braulke, T., Prata, M.J., Alves, S.

    المساهمون: Repositório Científico do Instituto Nacional de Saúde

    مصطلحات موضوعية: Lysosomal Storage Diseases, Mucolipidosis type II, RNA Therapies, Doenças Lisossomais de Sobrecarga, Terapias de RNA, Doenças Genéticas

    وصف الملف: application/pdf

    الاتاحة: http://hdl.handle.net/10400.18/6791

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  15. 15
    Conference
    From bench to bioterium and back again: development of a U1snRNA-based therapeutic strategy for mucopolysaccharidosis IIIC

    المؤلفون: Santos, Juliana Inês, Matos, Liliana, Oliveira, Paula Alexandra, Gonçalves, Mariana, Pires, Maria João, Coutinho, Maria Francisca, Prata, Maria João, Alves, Sandra

    مصطلحات موضوعية: Lysosomal Storage Diseases, RNA Therapies, Doenças Lisossomais de sobrecarga, Doenças Genéticas, Terapias de RNA

    Relation: http://hdl.handle.net/10400.18/7462

    الاتاحة: http://hdl.handle.net/10400.18/7462

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  16. 16
    Conference
    From bench to bioterium and back again: development of a U1snRNA-based therapeutic strategy for mucopolysaccharidosis IIIC

    المؤلفون: Santos, Juliana Inês Pinto, Matos, Liliana, Oliveira, Paula Alexandra, Gonçalves, Mariana, Pires, Maria João, Coutinho, Maria Francisca, Prata, Maria João, Alves, Sandra

    مصطلحات موضوعية: Lysosomal Storage Diseases, RNA Therapies, Doenças Lisossomais de sobrecarga, Terapias de RNA, Doenças Genéticas, Mucopolysaccharidoses type III

    Relation: http://hdl.handle.net/10400.18/7457

    الاتاحة: http://hdl.handle.net/10400.18/7457

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  17. 17
    Conference
    The 2020s tooth fairy: from loose tooth to neuronal cell cultures, a method to establish patient-derived MPS III neuronal cell lines

    المؤلفون: Coutinho, Maria Francisca, Santos, Juliana Inês, Matos, Liliana, Alves, Sandra

    مصطلحات موضوعية: Lysosomal Storage Diseases, MPS III, Therapeutic RNA Drugs, Dental Pulp Stem Cells, Patient-derived MPS III Neuronal Cell Lines, Doenças Genéticas, Terapias de RNA

    Relation: http://hdl.handle.net/10400.18/7461

    الاتاحة: http://hdl.handle.net/10400.18/7461

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  18. 18
    Conference
    Development of RNA based approaches to exploit alternative therapies for Lysosomal Storage Diseases

    المؤلفون: Matos, L., Santos, J.I., Rocha, M., Coutinho, M.F., Gaspar, P., Voltolini Velho, R., Braulke, T., Prata, M.J., Alves, S.

    مصطلحات موضوعية: Lysosomal Storage Diseases, Mucolipidosis type II, RNA Therapies, Doenças Lisossomais de Sobrecarga, Terapias de RNA, Doenças Genéticas

    Relation: http://hdl.handle.net/10400.18/6791

    الاتاحة: http://hdl.handle.net/10400.18/6791

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  19. 19
    Conference
    The use of a modified U1 snRNA as a therapeutic strategy to correct a 5’ splice-site mutation in Mucopolysaccharidosis IIIC: in vitro steps towards an in vivo approach

    المؤلفون: Matos, L., Santos, J.I., Rocha, M., Coutinho, M.F., Prata, M.J., Alves, S.

    مصطلحات موضوعية: Lysosomal Storage Disorders, RNA Therapies, Terapias de RNA, Terapias de Splicing, U1 snRNA, MPS IIIC, Doenças Lisossomais de Sobrecarga, Doenças Genéticas

    Relation: http://hdl.handle.net/10400.18/6792

    الاتاحة: http://hdl.handle.net/10400.18/6792

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  20. 20
    Conference
    Mutation-adapted U1snRNA as a therapeutic strategy for Mucopolysaccharidosis IIIC: in vitro and in vivo studies

    المؤلفون: Gonçalves, Mariana, Matos, Liliana, Santos, Juliana Inês, Coutinho, Maria Francisca, Prata, Maria João, Pires, Maria João, Oliveira, Paula, Alves, Sandra

    مصطلحات موضوعية: Doenças Genéticas, Genética Humana, Doenças Lisossomais de Sobrecarga, Terapias de RNA, MPS IIIC

    Relation: http://hdl.handle.net/10400.18/8194

    الاتاحة: http://hdl.handle.net/10400.18/8194

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