Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment

Judit Tomsen-Melero; Solène Passemard; Natalia García-Aranda; Zamira Vanessa Díaz-Riascos; Ramon González-Rioja; Jannik Nedergaard Pedersen; Jeppe Lyngsø; Josep Merlo-Mas; Edgar Cristóbal-Lecina; José Luis Corchero; Daniel Pulido; Patricia Cámara-Sánchez; Irina Portnaya; Inbal Ionita; Simó Schwartz; Jaume Veciana; Santi Sala; Miriam Royo; Alba Córdoba; Dganit Danino; Jan Skov Pedersen; Elisabet González-Mira; Ibane Abasolo; Nora Ventosa

doi:10.1021/acsami.0c16871

Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment

Judit Tomsen-Melero, Solène Passemard, Natalia García-Aranda, Zamira Vanessa Díaz-Riascos, Ramon González-Rioja, Jannik Nedergaard Pedersen, Jeppe Lyngsø, Josep Merlo-Mas, Edgar Cristóbal-Lecina, José Luis Corchero, Daniel Pulido, Patricia Cámara-Sánchez, Irina Portnaya, Inbal Ionita, Simó Schwartz, Jaume Veciana, Santi Sala, Miriam Royo, Alba Córdoba, Dganit DaninoJan Skov Pedersen, Elisabet González-Mira^*, Ibane Abasolo, Nora Ventosa

^*Corresponding author for this work

Biotechnology and Food Engineering

Research output: Contribution to journal › Article › peer-review

17 Scopus citations

Abstract

Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to overcome these problems is the use of nanocarriers for encapsulating the enzyme. Nanoliposomes functionalized with RGD peptide have already emerged as a good platform to protect and deliver GLA to endothelial cells. However, low colloidal stability and limited enzyme entrapment efficiency could hinder the further pharmaceutical development and the clinical translation of these nanoformulations. Herein, the incorporation of the cationic miristalkonium chloride (MKC) surfactant to RGD nanovesicles is explored, comparing two different nanosystems-quatsomes and hybrid liposomes. In both systems, the positive surface charge introduced by MKC promotes electrostatic interactions between the enzyme and the nanovesicles, improving the loading capacity and colloidal stability. The presence of high MKC content in quatsomes practically abolishes GLA enzymatic activity, while low concentrations of the surfactant in hybrid liposomes stabilize the enzyme without compromising its activity. Moreover, hybrid liposomes show improved efficacy in cell cultures and a good in vitro/ in vivo safety profile, ensuring their future preclinical and clinical development.

Original language	English
Pages (from-to)	7825-7838
Number of pages	14
Journal	ACS applied materials & interfaces
Volume	13
Issue number	7
DOIs	https://doi.org/10.1021/acsami.0c16871
State	Published - 24 Feb 2021

Keywords

Fabry disease
Miristalkonium chloride (MKC)
Nanovesicles
RGD targeting
α-galactosidase A (GLA)

Access to Document

10.1021/acsami.0c16871

Cite this

Tomsen-Melero, J., Passemard, S., García-Aranda, N., Díaz-Riascos, Z. V., González-Rioja, R., Pedersen, J. N., Lyngsø, J., Merlo-Mas, J., Cristóbal-Lecina, E., Corchero, J. L., Pulido, D., Cámara-Sánchez, P., Portnaya, I., Ionita, I., Schwartz, S., Veciana, J., Sala, S., Royo, M., Córdoba, A., ... Ventosa, N. (2021). Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment. ACS applied materials & interfaces, 13(7), 7825-7838. https://doi.org/10.1021/acsami.0c16871

@article{787d630b805d4a428751654b5cf6b208,

title = "Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment",

abstract = "Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to overcome these problems is the use of nanocarriers for encapsulating the enzyme. Nanoliposomes functionalized with RGD peptide have already emerged as a good platform to protect and deliver GLA to endothelial cells. However, low colloidal stability and limited enzyme entrapment efficiency could hinder the further pharmaceutical development and the clinical translation of these nanoformulations. Herein, the incorporation of the cationic miristalkonium chloride (MKC) surfactant to RGD nanovesicles is explored, comparing two different nanosystems-quatsomes and hybrid liposomes. In both systems, the positive surface charge introduced by MKC promotes electrostatic interactions between the enzyme and the nanovesicles, improving the loading capacity and colloidal stability. The presence of high MKC content in quatsomes practically abolishes GLA enzymatic activity, while low concentrations of the surfactant in hybrid liposomes stabilize the enzyme without compromising its activity. Moreover, hybrid liposomes show improved efficacy in cell cultures and a good in vitro/ in vivo safety profile, ensuring their future preclinical and clinical development.",

keywords = "Fabry disease, Miristalkonium chloride (MKC), Nanovesicles, RGD targeting, α-galactosidase A (GLA)",

author = "Judit Tomsen-Melero and Sol{\`e}ne Passemard and Natalia Garc{\'i}a-Aranda and D{\'i}az-Riascos, {Zamira Vanessa} and Ramon Gonz{\'a}lez-Rioja and Pedersen, {Jannik Nedergaard} and Jeppe Lyngs{\o} and Josep Merlo-Mas and Edgar Crist{\'o}bal-Lecina and Corchero, {Jos{\'e} Luis} and Daniel Pulido and Patricia C{\'a}mara-S{\'a}nchez and Irina Portnaya and Inbal Ionita and Sim{\'o} Schwartz and Jaume Veciana and Santi Sala and Miriam Royo and Alba C{\'o}rdoba and Dganit Danino and Pedersen, {Jan Skov} and Elisabet Gonz{\'a}lez-Mira and Ibane Abasolo and Nora Ventosa",

note = "Publisher Copyright: {\textcopyright} 2021 American Chemical Society",

year = "2021",

month = feb,

day = "24",

doi = "10.1021/acsami.0c16871",

language = "英语",

volume = "13",

pages = "7825--7838",

journal = "ACS applied materials & interfaces",

issn = "1944-8244",

publisher = "American Chemical Society",

number = "7",

}

Tomsen-Melero, J, Passemard, S, García-Aranda, N, Díaz-Riascos, ZV, González-Rioja, R, Pedersen, JN, Lyngsø, J, Merlo-Mas, J, Cristóbal-Lecina, E, Corchero, JL, Pulido, D, Cámara-Sánchez, P, Portnaya, I, Ionita, I, Schwartz, S, Veciana, J, Sala, S, Royo, M, Córdoba, A, Danino, D, Pedersen, JS, González-Mira, E, Abasolo, I & Ventosa, N 2021, 'Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment', ACS applied materials & interfaces, vol. 13, no. 7, pp. 7825-7838. https://doi.org/10.1021/acsami.0c16871

Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment. / Tomsen-Melero, Judit; Passemard, Solène; García-Aranda, Natalia et al.
In: ACS applied materials & interfaces, Vol. 13, No. 7, 24.02.2021, p. 7825-7838.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment

AU - Tomsen-Melero, Judit

AU - Passemard, Solène

AU - García-Aranda, Natalia

AU - Díaz-Riascos, Zamira Vanessa

AU - González-Rioja, Ramon

AU - Pedersen, Jannik Nedergaard

AU - Lyngsø, Jeppe

AU - Merlo-Mas, Josep

AU - Cristóbal-Lecina, Edgar

AU - Corchero, José Luis

AU - Pulido, Daniel

AU - Cámara-Sánchez, Patricia

AU - Portnaya, Irina

AU - Ionita, Inbal

AU - Schwartz, Simó

AU - Veciana, Jaume

AU - Sala, Santi

AU - Royo, Miriam

AU - Córdoba, Alba

AU - Danino, Dganit

AU - Pedersen, Jan Skov

AU - González-Mira, Elisabet

AU - Abasolo, Ibane

AU - Ventosa, Nora

PY - 2021/2/24

Y1 - 2021/2/24

N2 - Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to overcome these problems is the use of nanocarriers for encapsulating the enzyme. Nanoliposomes functionalized with RGD peptide have already emerged as a good platform to protect and deliver GLA to endothelial cells. However, low colloidal stability and limited enzyme entrapment efficiency could hinder the further pharmaceutical development and the clinical translation of these nanoformulations. Herein, the incorporation of the cationic miristalkonium chloride (MKC) surfactant to RGD nanovesicles is explored, comparing two different nanosystems-quatsomes and hybrid liposomes. In both systems, the positive surface charge introduced by MKC promotes electrostatic interactions between the enzyme and the nanovesicles, improving the loading capacity and colloidal stability. The presence of high MKC content in quatsomes practically abolishes GLA enzymatic activity, while low concentrations of the surfactant in hybrid liposomes stabilize the enzyme without compromising its activity. Moreover, hybrid liposomes show improved efficacy in cell cultures and a good in vitro/ in vivo safety profile, ensuring their future preclinical and clinical development.

AB - Fabry disease is a rare lysosomal storage disorder characterized by a deficiency of α-galactosidase A (GLA), a lysosomal hydrolase. The enzyme replacement therapy administering naked GLA shows several drawbacks including poor biodistribution, limited efficacy, and relatively high immunogenicity in Fabry patients. An attractive strategy to overcome these problems is the use of nanocarriers for encapsulating the enzyme. Nanoliposomes functionalized with RGD peptide have already emerged as a good platform to protect and deliver GLA to endothelial cells. However, low colloidal stability and limited enzyme entrapment efficiency could hinder the further pharmaceutical development and the clinical translation of these nanoformulations. Herein, the incorporation of the cationic miristalkonium chloride (MKC) surfactant to RGD nanovesicles is explored, comparing two different nanosystems-quatsomes and hybrid liposomes. In both systems, the positive surface charge introduced by MKC promotes electrostatic interactions between the enzyme and the nanovesicles, improving the loading capacity and colloidal stability. The presence of high MKC content in quatsomes practically abolishes GLA enzymatic activity, while low concentrations of the surfactant in hybrid liposomes stabilize the enzyme without compromising its activity. Moreover, hybrid liposomes show improved efficacy in cell cultures and a good in vitro/ in vivo safety profile, ensuring their future preclinical and clinical development.

KW - Fabry disease

KW - Miristalkonium chloride (MKC)

KW - Nanovesicles

KW - RGD targeting

KW - α-galactosidase A (GLA)

UR - http://www.scopus.com/inward/record.url?scp=85101512052&partnerID=8YFLogxK

U2 - 10.1021/acsami.0c16871

DO - 10.1021/acsami.0c16871

M3 - 文章

C2 - 33583172

AN - SCOPUS:85101512052

SN - 1944-8244

VL - 13

SP - 7825

EP - 7838

JO - ACS applied materials & interfaces

JF - ACS applied materials & interfaces

IS - 7

ER -

Impact of chemical composition on the nanostructure and biological activity of α‑galactosidase‑loaded nanovesicles for Fabry disease treatment

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Cite this