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Mol Ther ; 32(5): 1407-1424, 2024 May 01.
Artigo em Inglês | MEDLINE | ID: mdl-38429927

RESUMO

Maintaining functional adipose innervation is critical for metabolic health. We found that subcutaneous white adipose tissue (scWAT) undergoes peripheral neuropathy (PN) with obesity, diabetes, and aging (reduced small-fiber innervation and nerve/synaptic/growth-cone/vesicle markers, altered nerve activity). Unlike with nerve injuries, peripheral nerves do not regenerate with PN, and therefore new therapies are needed for treatment of this condition affecting 20-30 million Americans. Here, we validated a gene therapy approach using an adipocyte-tropic adeno-associated virus (AAV; serotype Rec2) to deliver neurotrophic factors (brain-derived neurotrophic factor [BDNF] and nerve growth factor [NGF]) directly to scWAT to improve tissue-specific PN as a proof-of-concept approach. AAVRec2-BDNF intra-adipose delivery improved tissue innervation in obese/diabetic mice with PN, but after longer periods of dietary obesity there was reduced efficacy, revealing a key time window for therapies. AAVRec2-NGF also increased scWAT innervation in obese mice and was more effective than BDNF, likely because Rec2 targeted adipocytes, the tissue's endogenous NGF source. AAVRec2-NGF also worked well even after 25 weeks of dietary obesity, unlike BDNF, which likely needs a vector that targets its physiological cellular source (stromal vascular fraction cells). Given the differing effects of AAVs carrying NGF versus BDNF, a combined therapy may be ideal for PN.


Assuntos
Adipócitos , Fator Neurotrófico Derivado do Encéfalo , Dependovirus , Terapia Genética , Vetores Genéticos , Obesidade , Gordura Subcutânea , Animais , Dependovirus/genética , Obesidade/terapia , Obesidade/metabolismo , Camundongos , Terapia Genética/métodos , Adipócitos/metabolismo , Vetores Genéticos/administração & dosagem , Vetores Genéticos/genética , Gordura Subcutânea/metabolismo , Fator Neurotrófico Derivado do Encéfalo/metabolismo , Fator Neurotrófico Derivado do Encéfalo/genética , Modelos Animais de Doenças , Fator de Crescimento Neural/metabolismo , Fator de Crescimento Neural/genética , Fatores de Crescimento Neural/metabolismo , Fatores de Crescimento Neural/genética , Técnicas de Transferência de Genes , Humanos , Masculino , Doenças do Sistema Nervoso Periférico/terapia , Doenças do Sistema Nervoso Periférico/etiologia , Doenças do Sistema Nervoso Periférico/metabolismo , Doenças do Sistema Nervoso Periférico/genética , Transdução Genética
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