Acknowledgements

We acknowledge the exacting experimentation performed by Dr. Ewa Sitnicka and Carl Storey for the murine stem cell/transplantation studies. We also thank Valerie Stepps and Casey Renée Haynes for their persistent, successful efforts, exploring the uptake of FITC-PMO by highly enriched CD34+ cells. Finally, the extensive effort made by Elizabeth Bartelmez to help prepare numerous NIH proposals is also gratefully acknowledged. These studies were primarily funded by NIH grants awarded to SB (NIH NIDDK, R01 DK48708; R43 HL093955 NIH/ NHLB; R43 HL093955 EY; RC1 EY020341 NIH/NEI; 1R44EY028070-01).

enhances multi-lineage repopulating efficiency. Journal of Stem Cell Research and Therapy.

Functional Activation of Autologous Human Diabetic Stem Cells for Cell Therapy

http://dx.doi.org/10.5772/intechopen.79650

[7] Sitnicka E, Ruscetti FW, Bartelmez SH. Transforming growth factor-β1 (TGFβ1) directly and reversibly inhibits the initial cell division of long-term repopulating stem cells. Blood.

[8] Yamazaki S, Ema H, Karlsson G, Yamaguchi T, Miyoshi H, et al. Nonmyelinating Schwann cells maintain hemtopoietic stem cell hibernation in bone marrow niche. Cell.

[9] Imbert AM, Bagnis C, Galindo R, Chabannon C, Mannoni P. A neutralizing anti-TGF-beta-1 antibody promotes proliferation oc CD34+Thy-1+ peripheral blood progenitors and increases the number of transduced progenitors. Experimental Hematology. 1998;26(5):374-381 [10] Karlsson G, Blank U, Moody JL, Ehringer M, Singbrant S, Deng CX, Karlsson S. Smad4 is critical for self-renewal of hematopoietic stem cells. The Journal of Experimental Medicine.

[11] Hudziak RM, Barofsky E, Barofsky DF, Weller DL, Huang SB, Weller DD. Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation. Antisense and

[12] Stein D, Foster E, Huang SB, Weller DD, Summerton J. A specificity comparison of four antisense types: Morpholino, 2'-O-methyl RNA, DNA, and phosphorothioate DNA. Anti-

[13] Summerton J, Review Article WDD. Morpholino antisense oligomers: Design, Prepatation, and properties. Antisense and Nucleic Acid Drug Development. 1997;7:187-195

[14] Ghosh C, Stein D, Dwight Weller D, Iversen P. Methods for evaluation of antisense mechanisms of action. In: Phillips I, editor. Antisense Technology Part A, General Methods, Methods of Delivery, and RNA Studies. Methods in Enzymology. Vol. 313.

[15] Giles RV, Spiller DG, Clark RE, Tidd DM. Antisense morpholino oligonucleotide analog induces missplicing of c-myc mRNA. Antisense and Nucleic Acid Drug Development.

[16] Ghosh C, Iversen PL. Intracellular delivery stategies for antisense phosphorodiamidate morpholino oligomers. Antisense & Nucleic Acid Drug Development. 2000;10(4):263-274

[17] Sazani P, Weller DL, Shrewsbury SB. Safety pharmacology and genotoxicity evaluation of AVI-4658. International Journal of Toxicology. 2010;29(2):143-156. DOI: 10.1177/

[18] Sazani P, Van Ness KP, Weller DL, Poage DW, Palyada K, Shrewsbury SB. Repeat-dose toxicology evaluation in cynomolgus monkeys of AVI-4658, a phosphorodiamidate morpholino oligomer (PMO) drug for the treatment of duchenne muscular dystrophy. International Journal of Toxicology. 2011;30(3):313-321. DOI: 10.1177/1091581811403505

2016;1(7):00045

1996;88:82-88

2011;147(5):1146-1158

2007;204(3):467-474

1999;9:213-220

1091581809359206

Nucleic Acid Drug Development. 1996;6:267-272

San Diego, CA: Academic Press; 1999. pp. 135-142

sense and Nucleic Acid Drug Development. 1997;7:151-157

#### Conflict of interest

The authors of this chapter have financial interest in BetaStem Therapeutics, Sausalito CA.
