预约演示

更新于：2025-05-07

PAPD5

更新于：2025-05-07

基本信息

别名

GLD4、Non-canonical poly(A) RNA polymerase PAPD5、PAP-associated domain-containing protein 5

+ [10]

简介

Terminal nucleotidyltransferase that catalyzes preferentially the transfer of ATP and GTP on RNA 3' poly(A) tail creating a heterogeneous 3' poly(A) tail leading to mRNAs stabilization by protecting mRNAs from active deadenylation (PubMed:21788334, PubMed:30026317). Also functions as a catalytic subunit of a TRAMP-like complex which has a poly(A) RNA polymerase activity and is involved in a post-transcriptional quality control mechanism. Polyadenylation with short oligo(A) tails is required for the degradative activity of the exosome on several of its nuclear RNA substrates. Doesn't need a cofactor for polyadenylation activity (in vitro) (PubMed:21788334, PubMed:21855801). Required for cytoplasmic polyadenylation of mRNAs involved in carbohydrate metabolism, including the glucose transporter SLC2A1/GLUT1 (PubMed:28383716). Plays a role in replication-dependent histone mRNA degradation, probably through terminal uridylation of mature histone mRNAs. May play a role in sister chromatid cohesion (PubMed:18172165). Mediates 3' adenylation of the microRNA MIR21 followed by its 3'-to-5' trimming by the exoribonuclease PARN leading to degradation (PubMed:25049417). Mediates 3' adenylation of H/ACA box snoRNAs (small nucleolar RNAs) followed by its 3'-to-5' trimming by the exoribonuclease PARN which enhances snoRNA stability and maturation (PubMed:22442037).

关联

项与 PAPD5 相关的药物

GSK3965193

靶点

PAPD5 x PAPD7

作用机制

PAPD5抑制剂 [+1]

在研机构

GSK Plc

原研机构

GlaxoSmithKline Pharmaceuticals Ltd.

在研适应症

慢性乙型肝炎

非在研适应症-

最高研发阶段临床1/2期

首次获批国家/地区-

首次获批日期1800-01-20

AB-161

靶点

PAPD5 x PAPD7

作用机制

PAPD5抑制剂 [+1]

在研机构

Arbutus Biopharma Corp.

原研机构

Arbutus Biopharma Corp.

在研适应症

乙型肝炎

非在研适应症-

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期1800-01-20

BCH-001

靶点

PAPD5

作用机制

PAPD5抑制剂

在研机构

The Children's Hospital Corp.

原研机构

The Children's Hospital Corp.

在研适应症

先天性角化不良

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期1800-01-20

项与 PAPD5 相关的临床试验

ACTRN12623000074695

/ Suspended临床1期

A Double-Blind, Randomized, Placebo-Controlled, Single Dose Study to Investigate the Safety, Tolerability, and Pharmacokinetics of AB-161 Following Oral Administration in Healthy Subjects

开始日期2023-03-16

申办/合作机构

Arbutus Biopharma Corp.

CTR20230241

/ Suspended临床1期

一项单中心、双盲、安慰剂对照的首次人体研究，用于评估单次和重复剂量给药 AK0706 片的安全性、耐受性、药代动力学特征及食物影响的 Ia 期临床试验

1、研究 AK0706 片在受试者中的安全性和耐受性，初步评估AK0706 对 QT 间期的影响。 2、评估健康受试者单次口服 AK0706 片和 HBsAg 阳性的 ALT 正常的受试者重复口服 AK0706 的 PK 特征。 3、评估食物对健康受试者口服 AK0706 片的 PK 特征的影响。 4、检测 AK0706 可能的代谢产物类型及主要代谢产物的 PK 特征（如适用）。

开始日期2023-02-16

申办/合作机构

厦门特宝生物工程股份有限公司

NCT05330455

/ Recruiting临床1/2期

Four-part, Randomized, Double-blind (Parts 1, 2A, 3 and 4), Multi-center, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GSK3965193 Monotherapy in Healthy Participants and in Participants Living With Chronic Hepatitis B Infection; and GSK3965193 in Combination With Bepirovirsen in Participants Living With Chronic Hepatitis B Infection

This Phase 1/2a multiple part study is a first time-in-human (FTIH) study designed to evaluate the safety, tolerability, and pharmacokinetics (PK) of single (Part 1) and repeat doses (Part 2) of GSK3965193 in healthy participants. Part 3 will evaluate the ability of GSK3965193 to lower hepatitis B virus surface antigen (HBsAg) in participants living with chronic hepatitis B infection (PLWCHB) and will be given the option to subsequently receive treatment with open label bepirovirsen. Part 4 will evaluate the safety and tolerability of combination therapy with GSK3965193 and bepirovirsen and the potential to effect sustained virologic response in PLWCHB.

开始日期2022-04-14

申办/合作机构

GSK Plc

100 项与 PAPD5 相关的临床结果

登录后查看更多信息

100 项与 PAPD5 相关的转化医学

登录后查看更多信息

0 项与 PAPD5 相关的专利（医药）

登录后查看更多信息

项与 PAPD5 相关的文献（医药）

2025-02-28·Global Health & Medicine

Clinical potential of SAG-524: A novel HBV RNA destabilizer with a unique mechanism of action

Article

作者: Tanaka, Yasuhito

SAG-524 is a novel, oral HBV RNA destabilizer developed to address the limitations of treatment with nucleos(t)ide analogues (NAs), which are effective against HBV DNA but show limited efficacy in reducing hepatitis B surface antigen (HBsAg) levels. SAG-524 exerts its effect by destabilizing HBV RNA by shortening the poly(A) tail, which leads to a significant reduction of both pgRNA and PreS/S mRNA. This destabilization seems to be specific for HBV RNA molecules. The mechanism involves the recruitment of PAPD5/7 by ZCCHC14 to the HBV RNA, where guanine is incorporated into the poly(A) tail to protect against degradation. SAG-524 disrupts this process by directly targeting PAPD5, thus destabilizing HBV RNA. In preclinical trials, oral administration of SAG-524 reduced serum HBsAg levels in HBV-infected PXB mice. When combined with NAs or capsid assembly modulators (CAMs), significant reductions in HBsAg, HBV DNA, and intrahepatic covalently closed circular DNA were observed. Safety studies conducted over 13 weeks in mice and monkeys revealed no significant toxicity, demonstrating the drug demonstrated a favorable safety profile. In conclusion, the novel mechanism of action, high oral bioavailability, and strong suppression of HBsAg make SAG-524 a promising candidate for future therapeutic use. The potential for combination therapy with NAs or CAMs underscores its capacity to contribute to achieving a functional cure for chronic HBV infection.

2024-12-01·International Immunopharmacology

Identification of RNA uridylation subtypes, and the prognostic and therapeutic value of RNA uridylation in systemic lupus erythematosus

Article

作者: Su, Sensen ; Ma, Zhanchuan ; Zhang, Yanli ; Yi, Huanfa ; Wu, Hao

The uridylation of 3'-RNA-a major process in epitranscriptomics- is catalyzed by terminal uridylyl transferases (TUTases), which are involved in multiple diseases and the immune response. Nonetheless, the role of TUTases in systemic lupus erythematosus (SLE) remains unknown. Here we identified increased level of MTPAP and ZCCHC6 and decreased level of PAPD5 and ZCCHC11 in SLE patients from Gene Expression Omnibus (GEO) GSE50772, GSE65391, and GSE121239. The random forest model was applied to screen 4 TUTase candidates (MTPAP, ZCCHC6, PAPD5, and ZCCHC11) to predict the susceptibility of SLE. A nomogram was constructed based on the 4 selected TUTase regulators. Decision curve analysis indicated that patients could benefit from the nomogram. Moreover, based on the 4 differentially expressed genes, individuals with SLE were divided into three patterns (Cluster A-C) using the consensus clustering method. Cluster B was enriched in adaptive immune cells, with the lowest TCE signature expression, manifesting a higher Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) than that in Cluster A and C. whereas, Cluster C was enriched in innate immune cells, with the highest T-cell exhaustion (TCE) signature expression, manifesting lower SLEDAI than that in Cluster B. Clinically, lupus nephritis (LN) patients manifested increased expression of MTPAP and ZCCHC6 and decreased expression of PAPD5 and ZCCHC11 in PBMCs using Quantitative Polymerase Chain Reaction (q-PCR). Immunohistochemistry (IHC) illustrated higher level of ZCCHC6 in the kidneys of LN patients than that in NC. In summary, TUTases could predict the occurrence of SLE and stratify patients based on their immune characteristics, eventually predicting the disease activity and guiding immune therapy.

2024-10-08·ACS Omega

Trypsin Digestion Conditions of Human Plasma for Observation of Peptides and Proteins from Tandem Mass Spectrometry

Article

作者: Chen, Zhuo Zhen ; Dufresne, Jaimie ; Marshall, John G. ; Miao, Ming ; Bowden, Peter

Previous meta-analysis indicated that plasma or serum proteome groups using various experimental conditions detected different peptides from the same plasma proteins, which is strong evidence for the veracity of blood fluid LC-ESI-MS/MS but also evidences that the trypsin digestion step is a key source of variation in plasma proteomics. Agreement between different digestion conditions and MS/MS algorithms may serve as an independent confirmation of the validity of the LC-ESI-MS/MS analysis of plasma peptides. Plasma contains a high percentage of albumin held together by multiple disulfide bonds; hence, reduction and/or alkylation may greatly enhance the digestion efficiency of albumin. Plasma proteins were precipitated in 90% acetonitrile, collected over quaternary amine resin, and eluted in NaCl prior to digestion treatments. To determine the effect of trypsin digestion methods, the plasma proteins were digested in 600 mM urea and 5% acetonitrile with trypsin alone, or reduced with 2 mM DTT followed by trypsin, or DTT followed by 15 mM iodoacetamide and then trypsin. The resulting peptides were analyzed by LC-ESI-MS/MS with a linear quadrupole ion trap (LIT). The MS/MS spectra were directly fit to peptides by the X!TANDEM and SEQUEST algorithms. Blank noise injections served as the analytical control, and 30 million random MS/MS served as the statistical control. Digesting human plasma with DTT reduction, or reduction and alkylation, resulted in a dramatic increase in the number and observation frequency of albumin peptides. In contrast, digestion with trypsin alone suppressed the observation of albumin, and instead, many low abundance plasma and cellular proteins showed higher observation frequency. Digestion with trypsin alone increased the observation frequency of APOC1, ACAN, ATRN, CPB2, GP2, GPX3, HBA1, PAPD5, PKD1, and many cellular proteins. After correction against noise and random controls, SEQUEST showed good agreement with the true positive plasma proteins identified by X!TANDEM and resulted in an R-squared of 0.5238 with an F-statistic of 10,930 on 9,935 protein gene symbols with a p-value < 2.2e-16. Digestion of plasma with trypsin alone avoids the complete digestion of albumin and permits the enhanced detection of some other cellular proteins from plasma. Different digestion approaches were complimentary and together resulted in a more comprehensive plasma proteome. The protein FDR q-values, the modest effect of background and Monte Carlo correction, and the significant STRING analysis were all consistent with the high fidelity of the rigorous X!TANDEM algorithm. In contrast, SEQUEST required significant correction against noise and statistical controls and selection of high cross correlation (XCorr) scores to show good agreement with X!TANDEM. There was qualitative and quantitative agreement between plasma proteins digested without alkylation from the orbital ion trap (OIT) versus the LIT instrument that showed highly significant regression against the X!TANDEM OIT monoisotopic results, those from heavy isotopes and other masses from X!TANDEM, and with those from MaxQuant. There was significant qualitative and quantitative agreement between the complementary digestion conditions consistent with the good fidelity of plasma analysis by LC-ESI-MS/MS with a sensitive linear ion trap.

项与 PAPD5 相关的新闻（医药）

2024-03-12

·药时代

【新药进展】全球乙肝新药进展(更新至2024年2月，独家整理)

编者按：为帮助大家更准确及时地获取全球乙肝新药最前沿信息，肝霖君结合2月Hepatitis B Foundation网站乙肝新药表单更新，查询各大药企官网更新的产品管线信息和相关网络资料中的乙肝新药报道做了系统整理，并汇总了慢乙肝新药与新药联合、新药与现有药物联合治疗的临床研究进展。本期表单更新有：治疗性疫苗TherVacB进入I期临床；新增处于临床前研究阶段的治疗性疫苗HBV vaccine、HBV RNA去稳定剂SAG-524以及表观遗传编辑药物TUNE-401。新药联合治疗的探索明显加速，包括新药与新药联合、新药与现有药物的联合。许多新药单药在I期或II期研究中未达到主要疗效目标，企业便积极开始了不同联合治疗方案的探索。这也将是未来乙肝全面临床治愈的必要策略。新药相关联合用药临床研究汇总部分新药进展1.TherVacB由德国Helmholtz Zentrum Muenchen研发的异源蛋白Prime/MVA增强治疗性乙肝疫苗TherVacB进入开放标签、剂量递增的Ia期临床研究（NCT05727267），旨在评估该产品在健康受试者中的安全性和免疫原性。2.HBV vaccineHBV vaccine是AstriVax研发的一款慢乙肝治疗性疫苗，可有效诱导特异性CD8+ T细胞，目前处于临床前研究阶段。3.SAG-524SAG-524是日本熊本大学研发的一款口服小分子HBV RNA去稳定剂，近期披露的临床前研究结果显示SAG-524呈剂量依赖性地降低HepG2.2.15细胞上清液中的HBV DNA（IC50 = 0.92 nM）和HBsAg（IC50 = 1.4 nM）；作用机制研究发现，SAG-524通过缩短poly（A）尾、特异性抑制PAPD5来破坏HBV RNA的稳定性。体内研究中，SAG-524有效降低HBV感染PXB小鼠的血清HBsAg和HBcrAg，最低有效剂量约为6 mg/kg/天；在猴子中服用高达1000 mg/kg/天、持续两周仍未产生明显毒性，安全性良好。4.TUNE-401TUNE-401是Tune Therapeutics研发的一款治疗慢性HBV感染的表观遗传编辑药物，其利用Tune的TEMPO平台从表观遗传学上沉默整合的HBV DNA以及cccDNA。临床前研究表明在原代人肝细胞中几乎完全抑制病毒DNA，持续时间超过550天；在人源化嵌合FRG小鼠模型中也观察到类似的高水平抑制。封面图来源：pixabay版权声明/免责声明本文为授权转载文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn默克终止BTK抑制剂开发！啃不动“自免”的骨头，肝损伤风险谁来验证……Ferring首席科学家杨翼博士：肥胖症疫苗离我们还有多远？礼来D药推迟上市，FDA的B面点击这里，欣赏更多精彩内容！

2023-03-08

·生物谷

Science：揭示USB1是一种调节造血发育的microRNA去腺苷化酶

在一项新的研究中，来自美国华盛顿大学和科罗拉多大学等研究机构的研究人员确定了一种潜在的治疗策略，用于治疗一种罕见的骨髓衰竭综合征。骨髓是骨骼内的海绵状组织，负责制造红细胞、白细胞和血小板。骨髓衰竭综合征会导致患危险性感染、贫血和血癌风险的增加。在一项新的研究中，来自美国华盛顿大学和科罗拉多大学等研究机构的研究人员确定了一种潜在的治疗策略，用于治疗一种罕见的骨髓衰竭综合征，即皮肤异色病合并中性粒细胞减少症（poikiloderma with neutropenia, PN）。这项新的研究也可能对治疗其他具有类似潜在功能障碍的骨髓衰竭综合征产生影响。相关研究结果发表在2023年3月3日的Science期刊上，论文标题为“USB1 is a miRNA deadenylase that regulates hematopoietic development”。 PN是由一个叫做USB1的基因的突变引起的。尽管知道了导致该疾病的遗传错误，但该错误导致骨髓衰竭的具体细节长期以来一直是个谜。当骨髓衰竭时，身体不能制造健康的红细胞、白细胞和血小板。患有这类疾病的人遭受感染的风险增加，容易患上皮肤癌和血癌。论文共同通讯作者、华盛顿大学圣路易斯医学院医学副教授Luis Batista博士说，“对于PN而言，目前没有治愈方法。患者面临着因感染并发症而死亡的高风险，科学家们不知道为什么这个基因的突变会导致骨髓衰竭。在这项新的研究中，我们发现了一种酶的新作用，为未来的临床试验打开了大门。有一些正在研究的药物可以阻断这种酶，所以我们希望治疗这些患者的临床医生可能会发现这是一个有前途的策略。” 这些作者研究了为模拟PN而设计的人类胚胎干细胞，发现microRNA分子的加工存在问题。这种加工问题导致特定的microRNA分子比正常情况下降解得更快。如果没有足够水平的这些microRNA，这些干细胞就不能产生正常的血细胞。论文共同第一作者、Batista实验室博士后研究助理Hochang Jeong博士说，“我们的研究显示，正常的USB1作为一种microRNA去腺苷化酶，切除这些microRNA的长尾巴，这稳定了它们的结构，使它们有时间完成形成血液产品的工作。当USB1在这种疾病中发生突变时，这些microRNA的尾巴比正常情况下更长。我们知道，拥有较长的尾巴会使microRNA和其他类别的RNA分子更容易成为降解的目标。我们了解到的情况是，在加长microRNA尾巴的酶和切除microRNA尾巴的酶之间应该有一个平衡。” 虽然目前还没有一种已知的方法来恢复正确切除microRNA尾巴的能力，但已经有研究性药物可以阻断负责切除microRNA尾巴的酶。在这种疾病中阻断这种酶可能会恢复microRNA尾巴的增加和减少之间的平衡。负责添加microRNA尾巴的酶被称为PAPD5和PAPD7，这些酶的抑制剂已经在包括乙型肝炎在内的其他疾病的人类临床试验中进行了研究。防止microRNA长尾巴的加入稳定了microRNA的结构，增加了它们的水平，并恢复了这些干细胞的正常血细胞形成。这些作者正在与行业伙伴合作，开发新的PAPD5和PAPD7抑制剂，专门用于治疗这种疾病和类似的疾病。 Batista说，“我们正在与不同的公司合作，开发更好和更特异性的PAPD5抑制剂来治疗这种罕见的骨髓衰竭综合征。在我的实验室里，我们是罕见疾病研究的大力倡导者。综合来看，罕见病一点都不罕见，这些患者值得我们关注。抑制PAPD5有望成为其他骨髓衰竭综合征的潜在治疗方法。”（生物谷 Bioon.com）参考资料： Ho-Chang Jeong et al. USB1 is a miRNA deadenylase that regulates hematopoietic development. Science, 2023, doi:10.1126/science.abj8379. 内容来源于网络，如有侵权，请联系删除。

临床研究

2023-03-07

·药明康德

前沿 | 被“放弃”的乙肝药物分子，却可用于治疗导致癌症的血液疾病

▎药明康德内容团队编辑说到攻克疾病，比起从零开始开发新疗法，重新挖掘现有药物的潜力，“老药新用”治疗目前无法被治愈的疾病或许更加能给人意外之喜。最近，来自华盛顿大学和科罗拉多大学的科学家们便在探究一种罕见性骨髓衰竭综合征时收获了这种惊喜，他们不仅揭示了这种疾病背后的病理机制，而且还发现一种曾经在临床试验中研究过的药物可以通过阻断一种关键酶来逆转这种缺陷。这篇研究近期以长文的形式发表在顶级学术期刊《科学》上。骨髓是骨骼内负责生成红细胞、白细胞和血小板的组织，如果骨髓发生病变，可能导致患者的造血能力异常，从而引发感染、贫血，甚至发展为癌症。而有这样的一群患者，由于先天性的基因突变不得不面对这些风险，他们便是皮肤异色病合并中性粒细胞减少症（PN）患者，目前临床上针对这种罕见病并没有治愈的方法。PN是由USB1基因突变引起的罕见病。早在1991年，医生们便在纳瓦霍族土著儿童中发现了一种罕见的综合征，这些患儿的胳膊和腿上出现了一种凸起的红色皮疹，呈圆形扩散，并随后出现色素沉着变化和皮肤表面可见的扩张血管。除了皮疹，患者还出现了白细胞减少，这使得他们容易受到致命感染，罹患皮肤癌和血癌的风险也更高。尽管科学家们已经知悉导致这种疾病的病症及其致病基因突变，但这种突变是如何导致骨髓衰竭的具体机制一直是个谜。现在，科学家们宣布已经破解了这个谜团，并找到了一种潜在的治疗药物。图片来源：123RF在这篇研究中，研究人员使用CRISPR-Cas9基因编辑技术在人类胚胎干细胞中制造了USB1基因突变。他们发现带有突变的干细胞外观和行为正常，便继续刺激它们转化为造血祖细胞。起初，USB1突变型细胞和对照细胞都按预期分化。但是在实验第16天后，研究小组注意到突变细胞中祖细胞的转化已经下降。经过一系列细胞实验和RNA测序分析后，研究人员揭示了其中的奥秘——血细胞分化所需的关键microRNA分子序列已经提前被破坏，没有足够水平的microRNA分子，干细胞就不能发育成正常的血细胞。图片来源：123RF在正常情况下，USB1基因编码的蛋白通过去除microRNA的分子的“尾巴”来稳定其结构。microRNA分子的尾巴也是一串核苷酸序列，这种序列传递给细胞的信号指示该microRNA分子应该被破坏。正如研究第一作者Ho-Chang Jeong博士的解释，USB1基因编码的“去尾”酶给了microRNA分子足够的时间来发挥它的作用。当它完成使命时，“加尾”酶PAPD5或PAPD7再将这种“尾巴”结构放回原处。但是当USB1基因发生突变，在没有这种“去尾”酶的情况下，microRNA分子在帮助祖细胞分化成血细胞之前就被破坏了，这会导致骨髓衰竭和随之而来的并发症。科学家们想要重新恢复“去尾”酶和“加尾”酶之间的平衡，并保护microRNA分子免于被破坏。虽然目前还没有已知的方法来恢复USB1突变细胞正确去除尾巴的能力，但已经有在研药物可以抑制为microRNA分子加上“尾巴”的酶活性。研究人员设想，在PN中阻断这种“加尾”酶可能会恢复平衡。于是他们测试了PAPD5抑制剂RG7834（也称为RO7020322）是否能有预期的效果。罗氏曾在临床试验中用这种药物治疗乙型肝炎，但最终罗氏放弃了该药物的开发。图片来源：123RF研究人员用RG7834处理了UB1突变型细胞，并研究它如何影响microRNA分子“尾巴”的添加。在施加这种药物后，研究人员观察到了细胞中无尾microRNA分子数量的增加，表明它有效遏制了microRNA分子的“加尾”过程、保护了这些分子免于被破坏。随后，研究人员继续评估了它是否也能恢复血细胞分化，而事实并没有让他们失望。经过RG7834的治疗后，突变型细胞中的中性粒细胞形成被显著改善，这表明RG7834有望成为PN的潜在治疗策略。最后，研究人员在USB1突变细胞中直接上调了microRNA水平，结果正如他们的预计——逆转microRNA水平后，血细胞的形成也被恢复，这进一步验证了他们的发现，通过抑制参与microRNA分子“加尾”作用的酶活性，可以逆转因USB1突变造成的血细胞异常，其作用等效于恢复被破坏的microRNA水平。据了解，主导该研究的科学家们现在正与业界合作伙伴合作，将他们的发现推向下一阶段。他们希望，这些发现不仅能为PN带来新的治疗方法，还能为其他拥有类似致病机制的骨髓综合征带来新的治疗方法。药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] Jeong, Ho-Chang et al. “USB1 is a miRNA deadenylase that regulates hematopoietic development.” Science (New York, N.Y.) vol. 379,6635 (2023): 901-907. doi:10.1126/science.abj8379[2] Possible treatment strategy identified for bone marrow failure syndrome，Retrieved March 7th, 2023 from https://medicine.wustl.edu/news/possible-treatment-strategy-identified-for-bone-marrow-failure-syndrome/[3] Drug once tested for hep b could help rare bone marrow syndrome，Retrieved March 7th, 2023 from https://www.fiercebiotech.com/research/drug-once-tested-hepatitis-b-could-help-patients-rare-bone-marrow-syndrome免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

基因疗法核酸药物