For example, a 3′ or 5′ UTR may be altered relative to a wild type or native UTR by the change in orientation or location as taught above or may be altered by the inclusion of additional nucleotides, deletion of nucleotides, swapping or transposition of nucleotides. Of particular importance is the delivery and function of a non-integrative polynucleotide. These UTRs or portions thereof may be placed in the same orientation as in the transcript from which they were selected or may be altered in orientation or location. In some embodiments, a modified oncology-related polynucleotide, oncology-related primary construct, or oncology-related mmRNA introduced to a cell may exhibit reduced degradation in the cell, as compared to an unmodified oncology-related polynucleotide, oncology-related primary construct, or oncology-related mmRNA. In another embodiment, the length may be sufficient to encode a peptide of amino acids, e. In one embodiment, the diblock copolymer may include PEG in combination with a polymer such as, but not limited to, polyethylenes, polycarbonates, polyanhydrides, polyhydroxyacids, polypropylfumerates, polycaprolactones, polyamides, polyacetals, polyethers, polyesters, poly orthoesters , polycyanoacrylates, polyvinyl alcohols, polyurethanes, polyphosphazenes, polyacrylates, polymethacrylates, polycyanoacrylates, polyureas, polystyrenes, polyamines, polylysine, poly ethylene imine , poly serine ester , poly L-lactide-co-L-lysine , poly 4-hydroxy-L-proline ester or combinations thereof.
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These sequences may be included at the beginning of the oncology-related polypeptide coding region, in the middle or at the terminus or alternatively into a flanking region.
PNAS 5: Tables 2 and 3 provide a listing of exemplary UTRs which may be utilized in the oncology-related primary construct of the present invention as flanking regions. In one embodiment, administration to the donor organism occurs either prior to any procedure to remove the organ or tissue or during removal.
The ester linkage can be internally located within the lipid chain or it may be terminally located at the 40 end of the lipid chain. As a non-limiting eutecitcs, the formulations may contain 1, 2, 3, 4 or 5 oncology-related mmRNA.
US9216205B2 – Modified polynucleotides encoding granulysin – Google Patents
In one embodiment, the post transcription control modulator may be a RNA regulatory sequence. Usually, the activity of the endogenous protein is deleterious to the subject, for example, do to mutation of the endogenous protein resulting in altered activity or localization.
In other embodiments, each of R 1 and R 2 is, independently, H, halo, hydroxy, thiol, optionally substituted alkyl, optionally substituted alkoxy, optionally substituted alkenyloxy, optionally substituted alkynyloxy, optionally substituted aminoalkoxy, optionally substituted alkoxyalkoxy, optionally substituted hydroxyalkoxy, optionally substituted amino, azido, optionally substituted aryl, optionally substituted aminoalkyl, optionally substituted aminoalkenyl, optionally substituted aminoalkynyl, or absent e.
When nucleic eutetics are provided with an IRES, further optionally provided is a second translatable region.
WO, WO and WO, each of which is herein incorporated by reference in its entirety. Microvasc Res A solid lipid nanoparticle SLN may be spherical with an average diameter between 10 to nm. As a non-limiting example, at least 5 nucleotides may be inserted at 1 location downstream of the transcription start site but upstream of the start codon and the at least 5 nucleotides may be the same base type.
The polymer may encapsulate the nanospecies or partially encapsulate the nanospecies. Either form of these residues may be present in the oncology-related polypeptides produced in accordance with the present invention. With the host of undesired consequences brought about by standard treatments such as chemotherapy and radiotherapy used today, genetic therapy for the manipulation of disease-related peptides provides a more targeted approach to disease diagnosis, treatment and management.
In particular embodiments, R 2 is optionally substituted alkoxy e. In other embodiments, the cosmetic polynucleotides, primary constructs or mmRNA include one of the following at the 2′ position: For example, provided are therapeutics containing one or more nucleic acids that encode trastuzumab and granulocyte-colony stimulating factor G-CSF.
USA1 – Modified polynucleotides encoding granulysin – Google Patents
In one embodiment, the modified mRNA encodes a polypeptide which acts as a radical scavenger or an immunosuppressive agent. The specific features can also be incorporated to ensure controlled down-regulation of the transcript in case they are misdirected to undesired organs sites.
Administration may be made by soaking, contact, injection, or by delivery to the blood of the donor or recipient. In some embodiments, each of R 1R eutectiscand R 3 is, independently, H, halo, hydroxy, thiol, optionally substituted alkyl, optionally substituted alkoxy, optionally substituted alkenyloxy, optionally substituted alkynyloxy, optionally substituted aminoalkoxy, optionally substituted alkoxyalkoxy, optionally substituted hydroxyalkoxy, optionally substituted amino, azido, optionally substituted aryl, optionally substituted aminoalkyl, optionally substituted aminoalkenyl, optionally substituted aminoalkynyl, or absent e.
In one embodiment, the fluids used during transplant may comprise compositions comprising modified mRNA.
As used herein, the “first region” may be referred to as a “coding region” or “region encoding” or simply the “first region. It is also possible to further characterize the 5′ and 3′ flanking regions by utilizing one or more available databases or algorithms.
Those described in U. Liposomes may contain a low or a high pH in order to improve the delivery of the pharmaceutical formulations.
US20140113960A1 – Modified polynucleotides encoding granulysin – Google Patents
Additional signal sequences which may be utilized in the present invention include those taught in, for example, databases such as those found at www. In one embodiment, the present invention provides methods of preparing a modified mRNA comprising at least one nucleotide e.
In some embodiments, R 15 is H, and R 16 is H or optionally substituted alkyl. In some embodiments, optionally substituted aminoalkyl is substituted with an optionally substituted sulfoalkyl or optionally substituted alkenyl.
Where these exist, they are shown in the tables as well. The N7- and 3’methlyated guanine provides the terminal moiety of the capped nucleic acid molecule e.