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Table 1.

CFTR genotypes of CF patients with no detectable Cl secretion (CFabsent) and residual Cl secretion (CFresidual) in native rectal epithelia.

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Figure 1.

1-EBIO activates CFTR-mediated basal and cholinergic Cl secretion in human rectal biopsies.

(A,B) Original recordings of effects of 1-EBIO (500 µM, basolateral) on basal and carbachol-induced (CCH) transepithelial voltage (Vte) and transepithelial resistance (Rte) across rectal biopsies from a control subject (A) and a CF patient carrying two severe CFTR mutations (R1158X/2183AA>G). (B) Experiments were performed in the presence of amiloride and indomethacin. Lumen-positive Vte responses reflect K+ secretion and lumen-negative responses reflect Cl secretion. Rte was determined from Vte downward deflections obtained by pulsed current injection. (C) Summary of effects of 1-EBIO on basal equivalent short-circuit current (Isc') in rectal biopsies from control subjects and CF patients with no detectable Cl secretion (CFabsent). (D,E) Effects of 1-EBIO on CCH-induced peak (open bars) and plateau (closed bars) Isc' responses in control (D) and CFabsent rectal tissues (E). (F,G) Effect of CFTRinh-172 on 1-EBIO-induced Cl secretion (lumen-negative Isc') under basal conditions (F) and on carbachol-induced (CCH) Cl secretion in the presence of 1-EBIO (G) in rectal biopsies from control subjects. Data are presented as mean±SEM. n = 7–13 individuals per group. * P<0.001 and P<0.01.

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Figure 2.

1-EBIO potentiates cAMP-mediated and cholinergic Cl secretion in human rectal biopsies and this effect is abrogated by inhibition of Ca2+-dependent K+ channels with clotrimazole.

(A) Original recording of effects of 1-EBIO (500 µM, basolateral) on cAMP-induced Cl secretion (IBMX/forskolin) and cholinergic co-activation (CCH), and effects of clotrimazole (30 µM, basolateral) on Cl secretory responses in a rectal biopsy from a control subject. Experiments were performed in the presence of amiloride, indomethacin and IBMX/forskolin. (B) Summary of effects of 1-EBIO on cAMP-induced Cl secretion and inhibition by clotrimazole in rectal tissues from control subjects. (C) Concentration-response curve for 1-EBIO-induced Cl secretion was determined in the presence of cAMP-mediated activation (IBMX/forskolin). (D) Effects of 1-EBIO on CCH-induced Cl secretion in the presence of IBMX/forskolin and inhibition by clotrimazole in control rectal tissues. Data are presented as mean±SEM. n = 17 individuals per group. *P<0.001. (E) RT-PCR analysis revealed transcripts of the clotrimazole-sensitive Ca2+-activated K+ channel KCNN4 in rectal biopsies from control and CF subjects. The 405 bp KCNN4 fragment was only identified in the presence (+), but not in the absence of reverse transcriptase (-).

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Figure 2 Expand

Figure 3.

1-EBIO mediated augmentation of cAMP-induced and cholinergic Cl secretion in human rectal biopsies does not depend on 293B-sensitive cAMP-dependent K+ channels.

(A) Original recording of effects of 1-EBIO (500 µM, basolateral) on cAMP-induced Cl secretion (IBMX/forskolin) and cholinergic co-activation (CCH), and effects of 293B (10 µM, basolateral) on Cl secretory responses in a rectal biopsy from a control subject. Experiments were performed in the presence of amiloride, indomethacin and IBMX/forskolin. (B, C) Summary of effects of 1-EBIO on cAMP-induced (B) and CCH-induced Cl secretion (C) in the absence and presence of 293B in rectal tissues from control subjects. Data are presented as mean±SEM. n = 19 individuals per group. *P<0.001. (D) RT-PCR analysis detected transcripts of the 293B-sensitive K+ channel KCNQ1 (728 bp fragment) in the presence (+), but not in the absence of reverse transcriptase (-), in rectal biopsies from control and CF subjects.

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Figure 3 Expand

Figure 4.

1-EBIO potentiates residual CFTR-mediated Cl secretion in CF rectal biopsies.

(A–C) Original recordings of effects of cAMP-mediated (IBMX/forskolin) and cholinergic (CCH) activation, and effects of 1-EBIO (500 µM, basolateral) on transepithelial voltage (Vte) and resistance (Rte) in rectal tissues from a control subject (A), a CF patient with no detectable Cl secretion (CFabsent; R1158X/2183AA>G) (B), and a CF patient with residual Cl secretion (CFresidual; F508del/Y161C), as evidence by lumen-negative Vte responses (C). Experiments were performed in presence of amiloride and indomethacin. 1-EBIO potentiated cAMP-mediated and cholinergic Cl secretion in control and CFresidual rectal tissues, but did not induce Cl secretion in the CFabsent tissue.

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Figure 4 Expand

Figure 5.

1-EBIO potentiates residual CFTR-mediated Cl secretion in CF rectal biopsies.

(A–F) Summary of effects of bumetanide (100 µM, basolateral) (A,B), CFTRinh-172 (20 µM, basolateral) (C,D) and 1-EBIO (500 µM, basolateral) (E,F) on cAMP-mediated (IBMX/forskolin) and cholinergic (CCH) activation of equivalent short circuit current (Isc') in rectal biopsies from control subjects, CF patients with no detectable Cl secretion (CFabsent) and CF patients with residual Cl secretion (CFresidual). All experiments were performed in the presence of amiloride and indomethacin. Only lumen-negative peak responses or plateau responses are shown for cholinergic (CCH) activation. Data are presented as mean±SEM. n = 4–26 individuals per group. *P<0.001, P<0.01 and P<0.05.

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Figure 5 Expand