LABELLING OF BIOMOLECULES
20220340946 · 2022-10-27
Inventors
- Robert NEELY (Birmingham, West Midlands, GB)
- Francisco FERNANDEZ-TRILLO (Birmingham, West Midlands, GB)
- Elodie JAGO (Birmingham, West Midlands, GB)
- Andrew WILKINSON (Birmingham, West Midlands, GB)
Cpc classification
C07H21/00
CHEMISTRY; METALLURGY
C07H1/00
CHEMISTRY; METALLURGY
International classification
Abstract
A method of reversibly labeling a biomolecule including providing a linker molecule (11) having a first functional group (LG) with a reactive center, a second functional group (FG) with a reactive center, and a cleavable, e.g. hydrolyzable, moiety (A-B-C). The method further includes forming a covalent bond between the biomolecule (10) and the reactive center of the first functional group (LG), forming a covalent bond between a first label (L1) and the reactive center of the second functional group (FG), cleaving the cleavable moiety (A-B-C), e.g. hydrolyzing the hydrolyzable moiety, of the linker molecule (11) to remove the first label (L1) and to form a third functional group (W) with a reactive center, and forming a covalent bond between a further molecule and the reactive center of the third functional group (W) to reform the cleavable moiety, e.g. hydrolyzable moiety (A-B-C).
Claims
1.-26. (canceled)
27. A method of reversibly labeling a biomolecule, the method comprising the steps of: a. providing a linker molecule, the linker molecule comprising a first functional group comprising a reactive center, a second functional group comprising a reactive center, and a cleavable moiety; b. forming a covalent bond between the biomolecule and the reactive center of the first functional group; c. forming a covalent bond between a first label and the reactive center of the second functional group; d. cleaving the cleavable moiety of the linker molecule to remove the first label and to form a third functional group comprising a reactive center; and e. forming a covalent bond between a further molecule and the reactive center of the third functional group to reform the cleavable moiety.
28. The method according to claim 27, wherein the further molecule comprises a fourth functional group, and wherein the method further comprises step f. forming a covalent bond between a second label and a reactive center of the fourth functional group.
29. The method according to claim 28, wherein the method further comprises step g. cleaving the cleavable moiety to remove the second label and to reform the third functional group.
30. The method according to claim 29, wherein the method further comprises step h. forming a covalent bond between a further molecule and the third functional group to reform the cleavable moiety, wherein the further molecule of step e. is optionally the same species as the further molecule of step h.
31. The method according to claim 27, wherein the further molecule comprises a second label.
32. The method according to claim 27, wherein the linker molecule comprises the following general formula: ##STR00030## wherein FG represents the second functional group comprising a reactive center; Z represents a non-reactive group of an aliphatic linkage or an aromatic linkage; A-B-C together represent the cleavable moiety; Y represents a non-reactive group of an aliphatic linkage or an aromatic linkage; LG represents a first functional group comprising a reactive center.
33. The method according to claim 32, wherein Z represents a polyether chain.
34. The method according claim 27, wherein the cleavable moiety A-B-C represents one of the following moieties: ##STR00031## ##STR00032## wherein R.sup.x represents a hydrogen atom, a deuterium atom, an aliphatic linkage, or an aromatic linkage.
35. The method according to claim 27, wherein the biomolecule is a polynucleotide.
36. The method according to claim 27, wherein step b. forming a covalent bond between the biomolecule and the reactive center of the first functional group further comprises the step of providing a catalyst.
37. The method according to claim 36, wherein the catalyst is an enzyme.
38. The method according to claim 27, wherein the linker molecule is an analogue of an S-adenosyl-1-methionine cofactor and has the following general formula: ##STR00033## wherein FG represents the second functional group; Z represents a non-reactive group of an aliphatic linkage or an aromatic linkage; A-B-C represent the cleavable moiety; Y represents a non-reactive group of an aliphatic linkage or an aromatic linkage; U represents an unsaturated bond comprising at least one of an alkene, an alkyne, an aryl group, a carbon atom comprising a carbonyl group, and a sulfur atom comprising one or two S═O bonds; k represents an integer of 1 or 2; and W.sup.− is a counter ion.
39. The method according to claim 38, wherein the linker molecule comprises an unsaturated moiety U.
40. The method according to claim 38, wherein the linker molecule has one of the following general formulas: ##STR00034## wherein the cleavable moiety is a Schiff base moiety comprising C═N—X—C-Q; p represents a number between 1 to 15; Q represents an oxygen atom, two hydrogen atoms, or one or more deuterium atoms independently bonded to the carbon center; X represents an oxygen atom or a nitrogen atom; Z represents a non-reactive group of an aliphatic linkage or an aromatic linkage; k represents an integer of 1 or 2; and FG represents the second functional group or ##STR00035## wherein the cleavable moiety is a Schiff base moiety comprising —C═N—N—C═O; p represents a number between 1 to 15; q represents a number between 1 to 15; k represents an integer of 1 or 2; and FG represents the second functional group. or ##STR00036## wherein the cleavable moiety is a Schiff base moiety comprising —C═N—O—; p represents a number between 1 to 15; wherein q represents a number between 1 to 15; k represents an integer of 1 or 2; and FG represents the second functional group.
41. The method according to claim 32, wherein the first functional group LG represents a halogen for reaction with an amino acid, a peptide, or a protein and/or the second functional group FG is an azide moiety.
42. The method according to claim 27, wherein forming a covalent bond between a first label and the reactive center of the second functional group comprises a reaction of an azide moiety with an alkyne moiety to form a carbon-nitrogen covalent bond.
43. The method according to claim 27, wherein cleaving the cleavable moiety of the linker molecule to remove a label and to form a third functional group comprises treatment with hydroxylamine.
44. The method according to claim 28, wherein at least one of the first label and the second label comprises at least one of a fluorescent molecule, a radioactive species, and a biological molecule.
45. The method according to claim 27, wherein forming a covalent bond between a further molecule and the third functional group in step e., to reform the cleavable moiety further comprises a reaction of an NH.sub.2 moiety with an aldehyde moiety on the further molecule to form a carbon-nitrogen double bond.
46. A method of reversibly labeling a polynucleotide molecule, the method comprising the steps of: a. providing a linker molecule (Compound A) having the following general formula: ##STR00037## wherein R represents a transferable group; FG represents the second functional group; Z represents a non-reactive group of an aliphatic linkage or an aromatic linkage; A-B-C represent the cleavable moiety; Y represents a non-reactive group of an aliphatic linkage or an aromatic linkage; U represents an unsaturated bond selected from the group consisting of an alkene, an alkyne, an aryl group, a carbon atom comprising a carbonyl group, and a sulfur atom comprising one or two S═O bonds; k represents an integer of 1 or 2; and b. forming a covalent bond between the polynucleotide molecule and the R group of Compound A using a DNA methyltransferase enzyme which is capable of using Compound A as a cofactor and under conditions that allow for the transfer of the R group of Compound A onto the polynucleotide molecule; c. forming a covalent bond between a first label and the second functional group FG of Compound A; d. hydrolyzing the cleavable moiety or hydrolyzable moiety of the linker molecule to remove the first label and to form an O-substituted hydroxylamine or an N-substituted hydrazone; e. forming a covalent bond between an aldehyde moiety of a further molecule and the O-substituted hydroxylamine or the N-substituted hydrazone to reform a Schiff base moiety.
Description
[0128] Embodiments of the invention will now be described by way of example only with reference to the accompanying drawings in which:
[0129]
[0130]
[0131]
[0132]
[0133]
[0134]
[0135]
[0136]
[0137]
[0138]
[0139]
[0140] Referring now to
[0141] There is shown a biomolecule 10 and a linker molecule 11. The linker molecule ii comprises a first functional group LG comprising a reactive centre, a second functional group FG comprising a reactive centre, a hydrolysable moiety (e.g. a Schiff base moiety) A-B-C, non-reactive groups Y and Z, and an unsaturated bond U. There is also shown a first label L1 and a second label L2.
[0142] The method of reversibly labelling the biomolecule 10 comprises the following steps, which are labelled on the schematic representation: [0143] a) providing a linker molecule 11, the linker molecule 11 comprising a first functional group LG comprising a reactive centre, a second functional group FG comprising a reactive centre, and a hydrolysable moiety, e.g. a Schiff base moiety, A-B-C; [0144] b) forming a covalent bond between the biomolecule 10 and the reactive centre of the first functional group LG; [0145] c) forming a covalent bond between the first label L1 and the reactive centre of the second functional group FG; [0146] d) hydrolysing the hydrolysable moiety A-B-C of the linker molecule 11 to remove the first label L1 and to form a third functional group W comprising a reactive centre; [0147] e) forming a covalent bond between a further molecule (not shown) and the reactive centre of the third functional group W to reform the hydrolysable moiety A-B-C.
[0148] Advantageously, the covalent bond formed between the reactive centre of the first functional group LG of linker molecule 11 and the biomolecule 10 adds chemical functionality to the biomolecule 10 in the form of the further functional group FG and the hydrolysable moiety A-B-C.
[0149] The method of the invention may further comprise the optional step i. of forming a covalent bond between a further molecule comprising a label, e.g. the second label L2, and the reactive centre of the third functional group W, to reform the hydrolysable moiety A-B-C.
[0150] In embodiments, step c. may be performed before step b.
[0151] Referring first to
[0152] Referring also to
[0153] There is shown a biomolecule 20 and the linker molecule 21. The linker molecule 21 may be either of those shown in
[0154] There is also shown a first label L1′ and a second label L2′.
[0155] In this embodiment, the biomolecule 20 is a DNA molecule and the linker molecule 21 is an analogue of the S-adenosyl-1-methionine cofactor.
[0156] The first functional group LG of the linker molecule 21A, 21B is S-adenosyl-I-homocysteine, the second functional group FG of the linker molecule is an azide moiety, the hydrolysable moiety is one of an hydrazone (Linker 21A) or an oxime (Linker 21B).
[0157] In this embodiment, hydrolysis of the hydrolysable moiety (e.g. Schiff base) A-B-C further comprises treatment with hydroxylamine, for example in an ammonium acetate buffer solution, e.g. at pH 4. The hydrolysable moiety A-B-C is hydrolysed to provide a third functional group W comprising a reactive centre; an N-substituted hydrazone (wherein the Schiff base is a hydroxylamine) or an O-substituted hydroxylamine (wherein the Schiff base is an oxime).
[0158] The further molecule 22 for use in step e. to form a covalent bond with the NH.sub.2 group of the N-substituted hydrazone (21A) or an O-substituted hydroxylamine (21B) to reform the hydrazone (21A) or the oxime (21B) Schiff base moiety is also shown in
[0159] In this embodiment, the functional group of the first label L1′ and the second label L2′ are both an alkyne moiety, which forms a C—N covalent bond with the reactive centre of the second functional group FG (an azide moiety) via a click chemistry reaction.
[0160] In this embodiment, step b. of the method 2 further comprises providing a catalyst, for example, an enzyme, e.g. a DNA methyltransferase enzyme capable of transferring an alkyl group from a S-adenosyl-1-methionine cofactor analogue.
[0161] The reversible and rewritable modification of DNA molecule 20 comprises the following steps, which are labelled on the schematic representation: [0162] a) providing a linker molecule 21A or 21B [0163] the linker molecule 21A or 21B comprising a S-Adenosyl-I-homocysteine moiety comprising a CH.sub.2-substituted trivalent sulphonium ion moiety LG, an azide moiety FG, and a hydrazone (21A) or an oxime (21B) or Schiff base moiety A-B-C; [0164] b) site-selective MTase-directed writing of DNA molecule 20 [0165] forming a covalent bond between the DNA molecule 20 (on one or more of a cytosine C5, cytosine N4 or adenosine N6) and the carbon of the CH.sub.2 group of the linker molecule 21A or 21B; [0166] c) modification of DNA molecule 20 via azide-alkyne cycloaddition [0167] forming a C—N covalent bond between the alkyne moiety of the first label L1′ and the azide of the linker molecule 21A or 21B; [0168] d) erasing introduced functionality via dynamic exchange [0169] hydrolysing the hydrazone (21A) or oxime (21B) Schiff base moiety of the linker molecule 21A or 21B to form N-substituted hydrazone (21A) or an O-substituted hydroxylamine (21B). [0170] e) re-writing the intermediate DNA molecule via Schiff-base formation [0171] forming a covalent bond between a further molecule (not shown) and the NH.sub.2 group of the N-substituted hydrazone (21A) or an O-substituted hydroxylamine (21B) to reform the hydrazone (21A) or the oxime (21B) Schiff base moiety.
[0172] Optionally, Step i. may involve further functionalising the DNA intermediate via standard conjugation techniques.
[0173] The further molecule, e.g. that of step e. or that of step i.; comprises an aldehyde moiety for reaction with the NH.sub.2 functionality of the third functional group to reform the Schiff base.
[0174] To further exemplify the invention, reference is also made to the following non-limiting Examples:
Synthesis of Precursor 1 for Use in the Synthesis of Linker 21A
[0175] Referring now to
Synthesis of Precursor 4 for Use in the Synthesis of Linker 21B
[0182] Referring now to
Example of a Linker Molecule for the Enzymatic Labelling of a Polynucleotide: MTase-Directed Labelling of a Polynucleotide
Synthesis of Linker Molecules 21A, 21B
[0191] Referring now to
3) Synthesis of S-adenosyl-1-methionine Cofactor Analogues 21A, 21B
[0192] 3.1 General coupling procedure [0193] Precursors 1, 4 were prepared and reacted with S-adenosyl-L-homocysteine under acidic conditions to give reversible and rewritable Boc-protected AdoMet derivatives. [0194] A solution of S-adenosyl-1-homocysteine (15 mg, 0.04 mmol) was made in a 1:1 mixture of formic and acetic acid (300 μl). Precursor 1 or 4 (tert-butyl 2-(8-bromooct-6-ynoyl)hydrazone-1-carboxylate or tert-butyl ((8-bromooct-6-yn-1-yl)oxy)carbamate) (1.2 mmol, 30 equivs) was then added dropwise, on ice. The reaction mixture was warmed to 35° C. and left to stir overnight. After overnight stirring the reaction mixture was extracted with diethyl ether and the aqueous layer was collected and dried by lyophilisation: MS: m/z [M+H]=638 (2), [M+H]=624 (5). [0195] 3.2 Cofactor Deprotection [0196] The AdoMet analogues were deprotected under acidic conditions to reveal the hydrazone or alkoxyamine moieties. [0197] The crude product was dissolved in TFA (400 μl) and left stir for 2 hrs at room temperature. After reaction the acid was removed under a flow of argon. [0198] 3.3 Cofactor Purification [0199] Any excess precursor was removed by purification. [0200] Both diastereomers of the deprotected cofactors could be separated by HPLC, a separation which was not possible at later stages. [0201] The crude reaction mixture was then dissolved in water (2 ml). Purification of AdoMet analogues was performed by preparative reversed-phase HPLC (ACE 5 C-18 25×2.12 cm) eluting with 20 mM Ammonium Acetate pH 5.5 Water (A)/MeOH (B) gradient at a flow rate of 10 ml/min. Gradient system: 30 mins 3-30% B, 30-97% B over 30 mins, hold at 97% B for 5 minutes, stop programme. Retention times: Hydrazide iso. 1=17.51 mins, iso. 2=18.73 mins, hydroxylamine iso. 1=25.47 mins, iso. 2=28.24 mins: MS: m/z [M+H]=538 (2), [M+H]=524 (5). [0202] The deprotected AdoMet derivatives slowly degrade, in particular following freeze-drying, via multiple pathways, giving additional peaks at higher retention times. [0203] 3.4 Aldehyde coupling [0204] To mitigate against degradation the AdoMet derivatives were reacted with a commercially available benzaldehyde immediately after purification by HPLC in order to minimise side reactions due to the nucleophilic nature of the hydrazone and alkoxyamine moieties. [0205] To the collected HPLC fractions Ald-PEG3-N3 (1.2 equivs) was added and rolled for 30 mins at room temperature. The fractions were then dried by lyophilsation. Once dry the solids were dissolved in 100 μl 0.1% Acetic Acid and stored at −20° C. Concentrations were determined by UV absorption analysis with E260=15.400 dm.sup.−3 mol.sup.−1 cm.sup.−1: MS: m/z [M+H]=867 (3), [M+H]=856 (6). [0206] The resulting linker molecules 21A and 21B contain reactive terminal azides that can be readily conjugated to a range if functional groups, while condensation of the aldehyde with the hydrazone or alkoxyamine incorporates a dynamic functionality, that can be reversibly functionalised. [0207] A slight excess of aldehyde (1.2 equivs) was employed to ensure full functionalisation of the deprotected intermediate. [0208] No degradation of the freeze-dried AdoMet derivatives 21A and 21B was observed.
MTase-Directed Labelling of a Polynucleotide with 21A and 21B
[0209] A restriction assay was used to demonstrate the activity of the MTases with the linker molecules 21A and 21B.
[0210] M.Taql (an N6-adenine DNA MTase) was incubated at 50° C. for 1 hour with a linker molecule 21A, 21B and plasmid pUC19, which has four recognition sites (TCGA) for the enzyme, see
[0211] Successful transfer of the functional group by M.Taql results in protection of the plasmid from restriction digestion by R.Taql, an endonuclease with the same target site as M.Taql.
[0212] Referring now to
[0213] In the absence of M.Taql-mediated alkylation (
[0214] Controls were run in the absence of AdoMet (
[0215] To test the labelling efficiency of each isomer, a cofactor dilution series was run to highlight any differences in affinity with the enzyme, with the second fraction having higher activity. The preferential isomer, diastereomer II, was carried forward for future experiments. Similar effects were seen with linker 21A. Both linker molecules 21A and 21B have the potential to be employed for the dynamic labelling of biomolecules.
[0216] M.Mpel is a cytosine-C5 MTase which targets the CpG dinucleotide. pUC19 was incubated with mutant M.Mpel (Q136A, N347A) and linker 21B before restriction enzyme R.Haell, which targets a subset of the CpG dinucleotides, was added. Efficient transalkylation of plasmid DNA with M.Mpel was also observed.
[0217] Evidence of the ability of MTases to alkylate DNA with linker 21A, 21B was achieved by targeting a 14 base pair oligonucleotide with one copy of M.Taql sequence (TCGA) for transalkylation. Labelling of the oligo was monitored directly using HPLC. Analysis was performed above the melting temperature of the DNA so that both strands could be clearly identified in the chromatogram. A clear shift in the retention time was seen upon labelling with linker 21A, 21B when compared to the retention times of the unmodified DNA. The shift was observed for both peaks, demonstrating that M.Taql was able to label both strands as a consequence of the palindromic nature of the sequence MTase recognizes. The shift was proportional to the size and nature of the linker transferred, with the AdoMet methylation resulting in a small shift in retention time and the oxime derivate 21B giving the biggest shift. The presence of a small amount of erased oligo DNA was observed and is likely due to hydrolysis under the HPLC conditions. Analysis of the individual peaks was carried out using MS which confirmed labelling was successful and the nature of the sidechain functionality introduced following incubation.
Reversible Enzymatic Labelling Protocol
[0218] The following protocols may be used in accordance with the embodiment shown in
Example of the Chemical Labelling of a Biomolecule: Cysteine Labelling of a Peptide
[0234] Referring now to
Synthesis of N-(2-(2-(2-(2-azidoethoxy)ethoxy)ethoxy)ethyl)-4-((2-(8-bromooct-6-ynoyl)hydrazoneylidene)methyl)benzamide 31
[0235] Activated linker (tert-butyl 2-(8-bromooct-6-ynoyl)hydrazone-1-carboxylate) 1 (20 mg, 0.06 mmol) was dissolved in 500 μL of TFA and stirred for 2 hours. TFA was evaporated to afford 14 mg of a yellow oil. This crude was then dissolved in 200 μL of PBS and Ald-Ph-PEG-Azide (22) (21 mg, 1 eq.) in solution in PBS was added (pH 7). Instantly, a white precipitate appeared. This white solid was filtered and dissolved in DCM for purification by HPLC preparative (0-60% ACN 40 min). MS: m/z [M+Na]=586.8/588.8 to afford linker molecule 31.
1) Chemical Labelling an Amino Acid (Step b of Method of the Invention)
[0236] A cysteine molecule 90 was labelled using linker molecule 31 in the following protocol.
[0237] Linker molecule 31 (1.2 mg) was dissolved in Ammonia 7M in Methanol. N-acetyl cysteine (Compound 90) (3 mg, 2.25 eq.) was added and mixture was stirred at room temperature for 2 hours. After reaction, compound 32 was formed, which was concentrated under pressure.
2) Schiff Base Reversal (Step c of Method of the Invention)
[0238] The Schiff base of compound 32 was hydrolysed in the following protocol. Compound 32 was dissolved in release buffer (release buffer: 231 mg NH.sub.2OH in 200 μl Ammonium buffer pH6) and heated at 50° C. for 1 hour. The products (compounds 33, 22) were purified by injection HPLC (analytical 0-60% ACN over 40 min and 100% ACN for 10 min).
[0239] The method of labelling an amino acid according to this Example of the invention shows that the method of the invention may be applied to label and release, and/or relabel, amino acids containing a thiol moiety other than cysteine. Moreover, this Example illustrates that peptides comprising amino acids containing a thiol moiety (e.g. cysteine) may be labelled and released, and relabeled, according to embodiments of the invention.
[0240] Advantageously, the method according to the invention is bio-orthogonal, that is, it may be performed in a biological system without interfering with the native biochemical processes. More advantageously, once reversed, the modification made to a biomolecule, e.g. a polynucleotide biomolecule, for example, DNA, is relatively small and hydrophilic meaning it will not affect the way that the biomolecule interacts in solution or with enzymes.
[0241] The linker molecule comprises a hydrolysable moiety, e.g. a Schiff base moiety, which is hydrolysable such that a label may be reversibly conjugated to the biomolecule. Advantageously, the conditions required to hydrolyse the Schiff base moiety are mild, which does not damage the structure of the biomolecule. Moreover, Schiff base chemistry is not commonly found in biomolecules, unlike prior art approaches such as the use of disulphide linkages. More advantageously, Schiff bases such as oximes and hydrazones are stable at physiological pH.
[0242] The covalent bonds formed using the linker molecule are reversible and rewritable. Advantageously, the label molecule may be used for repeated modifications of biomolecules, for example, labelling, capture, release, refunctionalisation for, e.g. fluorescent labelling, and/or imaging.
[0243] The analogue of S-adenosyl-1-methionine cofactor may be designed to comprise a linker molecule of any suitable structure.
[0244] Advantageously, the second functional group of the linker molecule is usable to further functionalise the biomolecule, e.g. polynucleotide, for example using click chemistry. This may be used for DNA capture, DNA complexation, drug attachment, and/or fluorescent labelling.
[0245] It will be appreciated by those skilled in the art that several variations to the aforementioned embodiments are envisaged without departing from the scope of the invention. For example, the biomolecule need not be a polynucleic acid. In embodiments, the first functional group of the linker molecule may be selected to be able to react with a moiety on a different biomolecule to form a covalent bond. For example, the biomolecule may comprise an azide moiety, and the first functional group may comprise an alkyne moiety, or vice versa, that is capable of forming a covalent bond via click chemistry.
[0246] It will also be appreciated by those skilled in the art that any number of combinations of the aforementioned features and/or those shown in the appended drawings provide clear advantages over the prior art and are therefore within the scope of the invention described herein.