%PDF-1.3 1 0 obj << /Type /Catalog /Outlines 2 0 R /Pages 3 0 R >> endobj 2 0 obj << /Type /Outlines /Count 0 >> endobj 3 0 obj << /Type /Pages /Kids [6 0 R 21 0 R 23 0 R 25 0 R ] /Count 4 /Resources << /ProcSet 4 0 R /Font << /F1 8 0 R /F2 9 0 R /F3 10 0 R /F4 11 0 R >> /XObject << /I1 12 0 R /I2 13 0 R /I3 20 0 R >> >> /MediaBox [0.000 0.000 595.280 841.890] >> endobj 4 0 obj [/PDF /Text /ImageC ] endobj 5 0 obj << /Creator (DOMPDF) /CreationDate (D:20220608155955-04'00') /ModDate (D:20220608155955-04'00') /Title (CRISPR Therapeutics: New Emerging Developments and Clinical Applications - IVT BLOG) >> endobj 6 0 obj << /Type /Page /Parent 3 0 R /Annots [ 14 0 R 16 0 R 18 0 R ] /Contents 7 0 R >> endobj 7 0 obj << /Length 6543 >> stream 1.000 1.000 1.000 rg 34.016 34.016 527.249 773.859 re f q 441.000 0 0 97.500 45.016 710.374 cm /I2 Do Q 0.000 0.000 0.000 rg BT 45.016 699.385 Td /F1 9.0 Tf [(Published on )] TJ ET BT 99.547 699.385 Td /F2 9.0 Tf [(IVT Network)] TJ ET BT 149.056 699.385 Td /F1 9.0 Tf [( \()] TJ ET BT 154.555 699.385 Td /F1 9.0 Tf [(http://www.ivtnetwork.com)] TJ ET 0.000 0.000 0.000 RG 0.18 w 0 J [ ] 0 d 154.555 698.234 m 259.081 698.234 l S BT 259.081 699.385 Td /F1 9.0 Tf [(\))] TJ ET 0.620 0.620 0.620 rg 45.016 686.888 506.159 0.750 re f 0.000 0.000 0.000 rg BT 45.016 662.142 Td /F3 15.0 Tf [(CRISPR Therapeutics: New Emerging Developments and Clinical Applications - )] TJ ET BT 45.016 644.329 Td /F3 15.0 Tf [(IVT BLOG)] TJ ET q 45.016 592.470 506.159 33.750 re W n 0.800 0.800 0.800 rg 45.016 625.845 13.005 0.750 re f 0.000 0.000 0.000 rg BT 45.016 605.856 Td /F1 9.0 Tf [(By)] TJ ET 0.800 0.800 0.800 rg 58.021 625.845 70.533 0.750 re f 0.000 0.000 0.000 rg BT 58.021 607.731 Td /F4 9.0 Tf [(Kaiser Jay Aziz)] TJ ET 0.800 0.800 0.800 rg 128.554 625.845 110.556 0.750 re f 0.000 0.000 0.000 rg BT 128.554 607.731 Td /F4 9.0 Tf [(Sep 24, 2021 7:00 am EDT)] TJ ET Q 0.463 0.443 0.443 rg BT 45.016 448.452 Td /F2 10.0 Tf [(This article was originally published in the Journal of Drug Delivery and Therapeutics, September 15, 2021, )] TJ ET BT 45.016 436.242 Td /F2 10.0 Tf [(Volume 11, Issue 5. It is being reprinted here with the permission of the author. IVT Network is republishing this )] TJ ET BT 45.016 424.032 Td /F2 10.0 Tf [(work to assist in bringing the latest research and developments to our audience, as well as to support the great )] TJ ET BT 45.016 411.822 Td /F2 10.0 Tf [(works of researchers like Kaiser Aziz.)] TJ ET 0.000 0.000 0.000 rg BT 45.016 388.170 Td /F4 12.0 Tf [(ABSTRACT)] TJ ET BT 45.016 374.943 Td 0.735 Tw /F1 10.0 Tf [(CRISPR gene editing is a genetic engineering technique applied in clinical applications in which the genomes of )] TJ ET BT 45.016 362.224 Td 0.286 Tw /F1 10.0 Tf [(living organisms may be modified. It is based on the principles of the CRISPR-Cas9 antiviral defense system. It is )] TJ ET BT 45.016 349.505 Td 0.376 Tw /F1 10.0 Tf [(based on delivering the Cas9 nuclease complexed with a synthetic guide RNA into a living organism cell and that )] TJ ET BT 45.016 336.787 Td 1.379 Tw /F1 10.0 Tf [(organisms genome can be cut and paste at a desired location, allowing existing genes to be modified for )] TJ ET BT 45.016 324.068 Td 2.062 Tw /F1 10.0 Tf [(desired outcome \(i.e., CRISPR for Precision Medicine\). CRISPR gene editing harnesses the natural defense )] TJ ET BT 45.016 311.349 Td 0.182 Tw /F1 10.0 Tf [(mechanisms of some bacteria to cut human DNA strands. Then the DNA strand either heals itself or injects a new )] TJ ET BT 45.016 298.630 Td 0.855 Tw /F1 10.0 Tf [(piece of DNA to mend the gap. Studies have been reported in Lung Cancer diagnosis and treatments. CRISPR-)] TJ ET BT 45.016 285.912 Td 3.737 Tw /F1 10.0 Tf [(based engineering techniques have been developed for T Cells and Stem cells applications \(i.e., Gene )] TJ ET BT 45.016 273.193 Td 0.268 Tw /F1 10.0 Tf [(Corrections in Hematopoietic Stem Cells for the Treatment of Blood and Immune System Diseases\). Even though )] TJ ET BT 45.016 260.474 Td 0.566 Tw /F1 10.0 Tf [(earlier CRISPR methodologies were used for performing simple DNA edits, recent applications include the ability )] TJ ET BT 45.016 247.755 Td 0.798 Tw /F1 10.0 Tf [(to delete genes or insert genes and edit regulatory regions in a wide range of cell types. The role of CRISPR in )] TJ ET BT 45.016 235.037 Td 0.216 Tw /F1 10.0 Tf [(human therapeutics is currently focused on utilizing CRISPR techniques to perform either in vivo editing of human )] TJ ET BT 45.016 222.318 Td 1.054 Tw /F1 10.0 Tf [(cellseverything from the head, eye all the way to neurons and liver cells--or performing ex vivo therapies. The )] TJ ET BT 45.016 209.599 Td 0.842 Tw /F1 10.0 Tf [(FDAs new genomic CRISPR technology based products approval process begins with review and evaluation of )] TJ ET BT 45.016 196.880 Td 2.152 Tw /F1 10.0 Tf [(preclinical studies in order to establish and characterize the proposed products safety profile. New genomic )] TJ ET BT 45.016 184.162 Td 0.414 Tw /F1 10.0 Tf [(products must be shown to be safe and effective for the FDA approval process. The sponsor of the new genomic )] TJ ET BT 45.016 171.443 Td 0.000 Tw /F1 10.0 Tf [(product must show that the product is safe and effective in human subjects.)] TJ ET BT 45.016 156.689 Td /F4 12.0 Tf [(INTRODUCTION)] TJ ET BT 45.016 143.462 Td 2.111 Tw /F1 10.0 Tf [(Pharmacogenomics \(PGx\), the development of how ones genes may affect an individuals response to new )] TJ ET BT 45.016 130.743 Td 2.072 Tw /F1 10.0 Tf [(treatments, has evolved in the translational stage of various specialties in medicine.)] TJ ET BT 456.871 130.743 Td 2.072 Tw /F1 10.0 Tf [( New developments )] TJ ET BT 45.016 118.024 Td 2.742 Tw /F1 10.0 Tf [(regarding Clustered Regularly Interspaced Short Palindromic Repeat \(CRISPR\) technology and its CRISPR )] TJ ET BT 45.016 105.305 Td 0.473 Tw /F1 10.0 Tf [(Associated proteins \(Cas9, Cas12a, Cas13\) essentially offer a programmable, sequence-specific means to target )] TJ ET BT 45.016 92.587 Td 0.322 Tw /F1 10.0 Tf [(the DNA or RNA sequence of your choice; however, Cas13 is a unique enzyme in that it targets RNA. The use of )] TJ ET BT 45.016 79.868 Td 1.488 Tw /F1 10.0 Tf [(multiple Cas nucleases \(i.e., Cas12a, Cas13a and Cas14a\) offers opportunity for a large range of sequences, )] TJ ET BT 45.016 67.149 Td 2.151 Tw /F1 10.0 Tf [(thus providing diagnostic information on a broader range of diseases. CRISPR technology comprises of two )] TJ ET BT 45.016 54.430 Td 0.777 Tw /F1 10.0 Tf [(components, a nuclease, e.g., Cas9, which acts like a pair of scissors and is responsible for cleavage of double )] TJ ET BT 45.016 41.712 Td 0.422 Tw /F1 10.0 Tf [(stranded DNA, and a single guide RNA, which forms a complex with the nuclease and guides it to the target site. )] TJ ET BT 45.016 28.993 Td 1.061 Tw /F1 10.0 Tf [(This technology can be designed to make a break at a specific target sequence in the DNA within a living cell, )] TJ ET 0.890 0.929 0.737 RG 1.5 w 0 J [ ] 0 d 64.516 466.470 138.000 117.750 re S q 136.500 0 0 116.250 65.266 467.220 cm /I3 Do Q BT 378.516 171.443 Td 0.000 Tw /F1 9.0 Tf [(1)] TJ ET BT 436.855 130.743 Td 2.072 Tw /F1 9.0 Tf [(2,3,4)] TJ ET endstream endobj 8 0 obj << /Type /Font /Subtype /Type1 /Name /F1 /BaseFont /Helvetica /Encoding /WinAnsiEncoding >> endobj 9 0 obj << /Type /Font /Subtype /Type1 /Name /F2 /BaseFont /Helvetica-Oblique /Encoding /WinAnsiEncoding >> endobj 10 0 obj << /Type /Font /Subtype /Type1 /Name /F3 /BaseFont /Times-Roman /Encoding /WinAnsiEncoding >> endobj 11 0 obj << /Type /Font /Subtype /Type1 /Name /F4 /BaseFont /Helvetica-Bold /Encoding /WinAnsiEncoding >> endobj 12 0 obj << /Type /XObject /Subtype /Image /Width 588 /Height 130 /Filter /FlateDecode /DecodeParms << /Predictor 15 /Colors 1 /Columns 588 /BitsPerComponent 8>> /ColorSpace /DeviceGray /BitsPerComponent 8 /Length 8715>> stream x]OK#MDA+x !\K ބ {o/|ygz&Y}Nt]]]UyD~|VVd`蓨CFAgy~u2W!Bm}u%2gSFDK_]惂la蓨-L}U)ϢKݯDFsA0_]恊W"+ Sk<`k
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